StemCell Therapy

In response to emailed questions, Cellino Biotech CEO and Co-founder Dr. Nabiha Saklayen, talked about the formation of the company and its goal to make stem cell therapies more accessible for patients.

Why did you start this company?

I see a huge need to develop a technology platform to enable the manufacture of cell therapies at scale. We recently closed a $16 million seed financing round led by Khosla Ventures and The Engine at MIT, with participation from Humboldt Fund. Cellino is on a mission to make personalized, autologous cell therapies accessible for patients. Stem cell-derived regenerative medicines are poised to cure some of the most challenging diseases within this decade, including Parkinsons, diabetes, and heart disease. Patient-specific cells provide the safest, most effective cures for these indications. However, current autologous processes are not scalable due to extensive manual handling, high variability, and expensive facility overhead. Cellinos vision is to make personalized regenerative medicines viable at large scale for the first time.

How did you meet your co-founders?

Nabiha Saklayen.

I met my co-founder Marinna Madrid in my Ph.D. research group. We had worked together for many years and had a fantastic working relationship. I then met our third co-founder Matthias Wagner through a friend. Matthias had built and run three optical technology companies in the Boston area and was looking to work with a new team. I was thrilled when we decided to launch the startup together at our second meeting. Matthias built the first Cellino hardware systems in what I like to call Matthias garage. In parallel, I was doing hundreds of expert interviews with biologists in academia and industry, and it started to narrow down our potential applications very quickly. Marinna was doing our first experiments with iPSCs. We iterated rapidly on building new versions of the hardware based on the features that were important to industry experts, such as single-cell precision and automation. Its incredible to witness our swift progress as a team.

What specific need or pain point are you seeking to address in healthcare/life sciences?

In general, autologous therapies are safer for patients because they do not require immunosuppression. The next iteration of cell therapies would use patient-specific stem cells banked ahead of time. Anytime a patient needs new cells, such as blood cells, neurons, or skin cells, we would generate them from a stem cell bank.

Today, patient-specific stem cell generation is a manual and artisanal process. A highly skilled scientist sits at a bench, looks at cells by eye, and removes unwanted cells with a pipette tip. Many upcoming clinical trials are using manual processes to produce stem cells for about ten to twenty patients.

At Cellino, we are converging different disciplines to automate this complex process. We use an AI-based laser system comes to remove any unwanted cells. By making stem cells for every human in an automated, scalable way, we are working towards our mission at Cellino to democratize personalized regenerative medicine.

What does your technology do? How does it work?

Cellinos platform combines label-free imaging and high-speed laser editing with machine learning to automate cell reprogramming, expansion, and differentiation in a closed cassette format, enabling thousands of patient samples to be processed in parallel in a single facility.

In general, autologous, patient-specific stem cell-derived therapies do not require immunosuppression and are safer for patients. Today, patient-specific stem cells are made manually, by hand. To scale the stem cell generation process, Cellino converges different disciplines to automate this complex process. We train machine learning algorithms to characterize cells before our AI-based laser system removes any unwanted cells. By making stem cells for every human in an automated, scalable way, our mission at Cellino is to democratize personalized regenerative medicine. Thats why our vision statement is Every human. Every cell.

Whats your background in healthcare? How did you get to where you are today?

When I arrived at Harvard University for my Ph.D. in physics, I wanted to be closer to real-world applications. Biology is inherently complex and beautiful, and I was interested in developing new physics-based tools to engineer cells with precision. During my Ph.D., I invented new ways to edit cells with laser-based nanomaterials. I collaborated with many brilliant biology groups at Harvard, including the Rossi, Scadden, and Church labs. Working closely with them convinced me that lasers offer a superior solution to editing cells with high precision. That realization compelled me to launch Cellino.

Do you have clinical validation for your product?

Our immediate goal for the next year is to show that our platform can produce personalized, high-quality, R&D-grade stem cells for different patients, which has not been established in an automated manner in the regenerative medicine industry so far. There is significant patient-to-patient variability in manual cell processing, which we eliminate with our platform.

Photo: Urupong, Getty Images

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Cellino Biotech developing tech to help scale stem cell therapies - MedCity News

Scribe and student: For the past five years, Brooke Ford has worked her way through college as an emergency room scribe at a few local hospitals. In her senior year at William & Mary, as the COVID-19 pandemic ravaged the nation, she managed medical scribes at two hospitals. Courtesy photo

by Adrienne Berard | April 16, 2021

For the better part of the past year, Brooke Fords return home from work has been an exercise in mitigating the spread of a potentially deadly pathogen.

She pulls off all her personal protective equipment, washes her hands, showers off, changes clothes, throws out any food or drink she may have touchedand greets her family.

I know a lot of people who were scared to even live with their families when they're working in a hospital room, because they didn't want to bring it home, said Ford, a William & Mary health sciences major who graduated in December.

For the past five years, Ford has worked her way through college as an emergency room scribe at a few local hospitals. In her senior year, as the COVID-19 pandemic ravaged the nation, she managed medical scribes at hospitals in Hampton and Newport News.

A scribe is responsible for recording the details of a patient's clinical history, current health issues, diagnoses and any medical procedures that are performed or prescribed, Ford explained. The scribe serves a vital role of being the ears and eyes in the room, so that a physician can focus squarely on treating the patient.

I lead teams at two hospitals, Ford said. During the pandemic, that has been really difficult because I had people that didn't want to come to work, which, I mean, it's scary. I dont blame them for that, but we had to learn to balance the fear and the need to do our jobs.

That balance between fear and the vital work ahead is one that has been familiar to healthcare professionals throughout the world this past year. For students graduating into the industry, the landscape has shifted dramatically, revealing fault lines in Americas healthcare system that these newest professionals are uniquely positioned to witness, study and address.

Matthew Tucker 22 started volunteering as an EMT with the Williamsburg Volunteer Fire Department his freshman year at William & Mary. Hes now the collegiate lieutenant, responsible for managing and training college students on the operations of emergency medical services provided by the local fire station.

I help train incoming college students on operations of the fire station, the ambulance, the equipment, all of those sorts of thing, he said.

This winter, as COVID-19 case numbers surged nationwide, Tucker was one of a handful of student volunteers who worked as first responders, supporting the career EMT staff at the fire station.

I know that the staff paramedics were especially burnt out. They couldnt trade shifts, they couldn't utilize their vacation time, so we were able to provide resources to support them in getting some relief, Tucker said. As students, we were able to see things that we haven't seen for 100 years. The amount of learning that can be done in that environment is astounding, and it has given me tremendous perspective on my future medical path.

Tucker said, as a student concentrating in public health, hes been particularly aware of the health disparities in the community he serves. It was immediately clear to him that the vast majority of emergency calls were coming from low-income neighborhoods.

I believe that we're often responding to places where the healthcare system has failed, where we are their primary care provider, a call to 911, taking them to the emergency room, he said. These disparities have always been there, but we're seeing them very plainly now, how socioeconomic status, job accessibility, food insecurity, all these different things affect someone's risk to COVID and ultimately affect people's health status. Weve not always been in tune to seeing that before the pandemic.

Sophie Kopec 21 spent this past summer analyzing data on experimental, early-stage clinical trials for the cell and gene medicine division of the non-profit Alliance for Regenerative Medicine. Explaining what she does is a key part of the job.

Its such an up-and-coming field that there are negative trigger words, all this stigma, especially around cell therapy, she said. So, part of my job was to look at data from all of these regenerative medicine clinical trials to identify the most promising ones. Then we translate that data into the commercial field to help show consumers that this is real, valid, verified science. Its not some kind of scam.

Kopec describes regenerative medicine as using the body's own functions to train it to fix itself.

For example, take gene therapy, in that case you have editing technology like CRISPR, where you can go in and actually edit the bodys genes and reprogram them to respond to the treatment and then body essentially heals itself, she said.

Kopec entered William & Mary with her sights hard-set on chemistry. It wasnt until her junior year, when she was working her way down the list of general education requirements, that she enrolled in a Foundations of Epidemiology course with Assistant Professor of Kinesiology and Health Sciences Carrie Dolan.

After taking that class, everything changed and I shifted my focus to public health, Kopec said. Ill always be grateful to William & Mary for that. I came into school with tunnel vision. I was going to go to med school and work in scrubs or in a lab, but my education broadened my perspective. Actually, it did more than that, it revealed my passion to me. Had I not been forced off track by the general education requirements, I would have never found this incredible spark and pursued this passion that I have now.

Graduation can be an anxiety-inducing milestone, even in the best of times. For those entering careers in the healthcare industry, the uncertainty of the pandemic has added another layer of stress and hope for overcoming the challenges of the past.

Next month, Kopec starts her new job as a clinical research coordinator at Children's National Hospital in D.C. Shell be serving in the neuro-oncology unit, working with children who have not responded to traditional therapy measures and providing experimental treatment that is often the last and only resort left. Her first in-person day at the hospital is scheduled for June 1.

Well be doing clinical trials that offer more therapeutic approaches to treating these late-stage tumors, she said. Even if the end result for some of these patients may be death, at least we know that we did everything in our power to help them and give them another chance to keep fighting and have another opportunity at life.

When Ford began her studies at William & Mary, she was interested in emergency medicine. She wanted to learn to respond in crisis to keep people alive. Now, after all she has witnessed in this year of pandemic, she hopes to study palliative care. She wants to learn how to help people live and die with dignity.

In medical school, they teach you how to save people, but what happens when you cant save them, when there is nothing else you can do? she said. Its not a conversation were often prepared for, but its so important. In a lot of ways, how can you know how you want to live if you don't know how you want to die?

So many families this year have had to make those choices for family members and they don't know what choices those family members would want to make for themselves, she added. Theyve had to make those choices, because those conversations haven't been had. I want to be there to help have those hard conversations.

After graduating from William & Mary, Tucker plans to go to medical school to obtain Master of Public Health and Medical Doctor degrees. He plans to work primarily with people experiencing homelessness and be able to treat them holistically.

If theres one main thing that I think that weve all learned this pandemic, its that health is incredibly complex, Tucker said. Truly, everything that we do impacts our health, every societal factor, institutional factor, personal factor that's put into place influences somebodys health.

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Lessons learned: W&M students working in healthcare reflect on a year of pandemic - William & Mary News

MIAMI--(BUSINESS WIRE)-- Organicell Regenerative Medicine, Inc. (OTCMKTS: BPSR), a clinical-stage biopharmaceutical company dedicated to the development of regenerative therapies, today announced that it has entered into an agreement with Oklahoma State University to evaluate Zofin for the treatment of respiratory diseases caused by virus infections of pandemic potential. This study site follows the Companys earlier announcement of the agreement reached with the Centers for Disease Control and Prevention (CDC) to conduct research to determine the anti-inflammatory and anti-infective effectiveness of Zofin in experimental models of influenza infection.

The ongoing COVID-19 pandemic has infected over 5 million people globally and caused over 300,000 case fatalities in 188 countries. Additionally, the CDC estimates between 39-55 million influenza related illnesses and more than 60,000 deaths due to influenza. The observed illnesses and case fatalities are primarily due to the lack of known effective antivirals and prophylactic vaccines to attenuate the viruses.

In this agreement, Organicell will supply Oklahoma State University with its lead compound, Zofin, which is an acellular material derived from human amniotic fluid. This extracellular vesicle-derived nanoparticle-based therapeutic will be assessed on its ability to induce antiviral and/or immunomodulatory activity against virus infections of pandemic potential as there is an unmet need for non-toxic and effective therapeutic approaches to deal with current and imminent pandemics.

Organicell is dedicated to continuing regenerative therapy research for diseases with unmet needs. We are thrilled to be partnering with Oklahoma State University and the CDC on the study of Zofin for viral infections of pandemic proportions. A year into the pandemic has shown us the importance of research aimed at the development of biologics, said Albert Mitrani, CEO of Organicell.

We are excited to partner with Oklahoma State University and the CDC to investigate how the extracellular vesicle-derived nanoparticles, in Zofin, will impact antiviral and/or immunomodulatory activity of viral infections with pandemic potential. Dr. Mari Mitrani, Chief Science Officer of Organicell.

About Zofin:

Zofin is an acellular biologic therapeutic derived from perinatal sources and is manufactured to retain naturally occurring microRNAs, without the addition or combination of any other substance or diluent. This product contains over 300 growth factors, cytokines, and chemokines as well as other extracellular vesicles/nanoparticles derived from perinatal tissues. Zofin is currently being tested in a phase I/II randomized, double blinded, placebo trial to evaluate the safety and potential efficacy of intravenous infusion of Zofin for the treatment of moderate to SARS related to COVID-19 infection vs placebo.

ABOUT ORGANICELL REGENERATIVE MEDICINE, INC.

Organicell Regenerative Medicine, Inc. (OTCMKTS: BPSR) is a clinical-stage biopharmaceutical company that harnesses the power of exosomes to develop innovative biological therapeutics for the treatment of degenerative diseases. The Companys proprietary products are derived from perinatal sources and manufactured to retain the naturally occurring exosomes, hyaluronic acid, and proteins without the addition or combination of any other substance or diluent. Based in South Florida, the company was founded in 2008 by Albert Mitrani, Chief Executive Officer and Dr. Mari Mitrani, Chief Scientific Officer. To learn more, please visit https://organicell.com/.

FORWARD-LOOKING STATEMENTS

Certain of the statements contained in this press release should be considered forward-looking statements within the meaning of the Securities Act of 1933, as amended (the Securities Act), the Securities Exchange Act of 1934, as amended (the Exchange Act), and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are often identified by the use of forward-looking terminology such as will, believes, expects, potential or similar expressions, involving known and unknown risks and uncertainties. Although the Company believes that the expectations reflected in these forward-looking statements are reasonable, they do involve assumptions, risks and uncertainties, and these expectations may prove to be incorrect. We remind you that actual results could vary dramatically as a result of known and unknown risks and uncertainties, including but not limited to: potential issues related to our financial condition, competition, the ability to retain key personnel, product safety, efficacy and acceptance, the commercial success of any new products or technologies, success of clinical programs, ability to retain key customers, our inability to expand sales and distribution channels, legislation or regulations affecting our operations including product pricing, reimbursement or access, the ability to protect our patents and other intellectual property both domestically and internationally and other known and unknown risks and uncertainties, including the risk factors discussed in the Companys periodic reports that are filed with the SEC and available on the SECs website (http://www.sec.gov). You are cautioned not to place undue reliance on these forward-looking statements All forward-looking statements attributable to the Company or persons acting on its behalf are expressly qualified in their entirety by these risk factors. Specific information included in this press release may change over time and may or may not be accurate after the date of the release. Organicell has no intention and specifically disclaims any duty to update the information in this press release.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210414005207/en/

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Organicell and Oklahoma State University Enter Into Agreement To Study Zofin in Respiratory Diseases Caused By Virus Infections Of Pandemic Potential...

Newswise A Phase II clinical trial to assess mesenchymal adult stem cells as a disease-modifying therapy for Parkinson's disease has been launched at The University of Texas Health Science Center at Houston (UTHealth).

"Studies have shown mesenchymal stem cells can migrate to the sites of injury and respond to the environment by secreting several anti-inflammatory and growth factor molecules that can restore tissue equilibrium and disrupt neuronal death," said Mya C. Schiess, MD, professor in the Department of Neurology and director and founder of the movement disorder subspeciality clinic and fellowship program at McGovern Medical School at UTHealth. "The stem cells interact directly with the immune cells, leading to an anti-inlammatory state that allows a restorative process to take place."

Safety and tolerability results, assessed on a previous trial, were recently published in the journal Movement Disorders. The Phase I study showed that there were no serious adverse reactions related to the stem cell influsion and no immunological reactions to the cells, which come from the bone marrow of a healthy adult donor. The study enrolled 20 patients with mild to moderate disease, who were infused with one of four different dosages and monitored for a year. Additionally, researchers reported a reduction in preripheral inflammatory markers and a reduction in motor symptoms.

Parkinson's diease is the second most common neurodegenerative disease, affecting more than a million Americans. It is also the fastest-growning of the neurodegenerative diseases, with more than 60,000 new cases identified every year. It is predicted that by 2040, Parkinson's disease will affect 17.5 million people worldwide.

Research has shown that one of the forces playing a critical role in the diease's development and progression is a chronic neuroinflammatory process that damages the brain's microenvironment and alters its healthy equilibrium. Inflammatiion perpetuates the neurodegenration in the brain areas that control movement, causing the tremors, imbalance, loss of speech, slowness, and other motor impairments.

The randomized, double-blind, placebo-controlled Phase II trial will investigate the safest and most effective number of repeat doses of stem cells to slow the progression of Parkinson's disease. The study will enroll 45 patients, ages 50 to 79, who will receive three infusions of either placebo or stem cell therapy at three-month intervals and will be followed for a year after the last infusion.

"Currently, there is no approved therapy that can delay the degenerative process in Parkinson's disease," Schiess said. "By investigating a treatment that can slow or stop the progression, we hope to improve the quality of life of those suffering from the disease. The ultimate goal is to use this treatment in individuals with a prodromal condition, meaning they are showing early signs of Parkinson's disease but are not yet clinically symptomatic. We hope to be able to potentially stop the diease's conversion or clinical manifestation in patients who are high-risk."

The Phase II trial, approved by the U.S. Food and Drug Administration, is supported with funding from the Michael J. Fox Foundation, John S. Dunn Foundation, and John and Kyle Kirksey.

Other McGovern Medical School faculty co-authors on the paper included Jessika Suescun, MD, Christopher Adams, MD, and Sean Savitz, MD, in the Department of Neurology. Marie-Francoise Doursout, PhD, Department of Anesthesiology; Charles Green, PhD, Department of Pediatrics; and Jerome G. Saltarrelli, PhD, Department of Surgery. Timothy M. Ellmore, PhD, Department of Psychology at the City College of New York, N.Y., was senior author.

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First in the nation, FDA-approved Phase II mesenchymal stem cell therapy for Parkinson's disease begins - Newswise

Myrtle Beach, SC - QC Kinetix (Myrtle Beach) takes a different healthcare approach to most clinics in Myrtle Beach. It is a regenerative medicine clinic that focuses on non-surgical methods of managing pain. Regenerative medicine focuses on whole-body healing by using blood, cells, and tissues to help repair damaged areas and lessen inflammation and pain. Whether the pain is orthopedic or deep in the muscles and tendons, the clinics regenerative medicine technique is a non-invasive way to heal the body.

The physicians at QC Kinetix (Myrtle Beach) believe that whether a patient is suffering from persistent pain caused by an injury, disease, or age can permanently solve the problem. They do this by focusing on several different medical methods to help their patients regain their lives pain-free, to perform daily tasks without difficulty. The clinic treatments include the use of lasers, ultrasounds, regenerative cells, and platelet-rich plasma.

For the success of platelet-rich plasma injections or PRP therapy, the team mixes the patients platelets with plasma before injecting it into the injured area. Another area of regenerative therapy methods the clinic uses is called stem cell therapy. The stem cell therapy technique involves the team taking stem cells from a patients bone marrow and injecting them into inflamed or damaged areas. This injection of stem cells prompts the body to generate more red blood cells, reducing inflammation and pain.

QC Kinetix (Myrtle Beach) guarantees that all the patients who trust them will feel increased pain relief from one of their regenerative medicine treatments. The pain clinic ensures that they alleviate pain on several parts of the body, including the shoulder, elbow, wrist, ankle, low back, hip, knee, and feet.

To alleviate the pain from these areas, the clinic offers an array of services for Myrtle Beach residents. They include QC Knee that treats knee pain, cracking/popping, knee arthritis, and injuries such as torn ACL, MCL, LCL, and meniscus. QC Injury for patients of acute sports-related and musculoskeletal injuries and joint pain. QC 2M takes care of joint pain due to arthritis in the knees, shoulder, ankle, wrist, feet, hands, elbow, lower back, hips, and more.

Patients who visit the Myrtle Beach knee pain doctoralso enjoy QC Per4M, a treatment to improve energy, muscle strength, endurance, and appropriate weight loss with hormone replacement therapy. And QC Medical for patients with non-sports-related musculoskeletal pain resulting from sciatica, plantar fasciitis, or tendonitis.

The entire QC Kinetix staff (Myrtle Beach) believes that regenerative medicine, including stem cell therapy, works on several conditions. They include arthritis, plantar fasciitis, muscle damage, orthopedic pain, and tendon issues all over the body.

To learn more about QC Kinetix (Myrtle Beach) - Myrtle Beach, joint pain doctor, call (843) 310-2703 to schedule an appointment. Or visit their location, 8210 Devon Ct Suite A, Myrtle Beach, SC, 29572, US. For any inquiries about the services they offer, visit their website for more information.

Media ContactCompany Name: QC Kinetix (Myrtle Beach)Contact Person: Adam RoseEmail: Send EmailPhone: (843) 310-2703Address:8210 Devon Ct Suite A City: Myrtle BeachState: SCCountry: United StatesWebsite: https://qckinetix.com/myrtle-beach/

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QC Kinetix (Myrtle Beach) is the Stem Cell Therapy and Regenerative Medicine Clinic Myrtle Beach - Press Release - Digital Journal

The Regenerative Medicine Market report is a compilation of first-hand information, qualitative and quantitative assessment by industry analysts, inputs from industry experts and industry participants across the value chain. The report provides in-depth analysis of parent market trends, macro-economic indicators and governing factors along with market attractiveness as per segments. The report also maps the qualitative impact of various market factors on market segments and geographies.

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Top LeadingCompaniesof Global Regenerative Medicine Market areDePuy Synthes, Osiris Therapeutics, Medtronic, Stryker, Acelity, ZimmerBiomet, UniQure, MiMedx Group, Cellular Dynamics International, Organogenesis, Vericel Corporation, Mesoblast, Guanhao Biotech, Vcanbio, Cytori, Golden Meditech, Bellicum Pharmaceuticals, Celgene, Gamida Cell and others.

On The Basis Of Product, The Regenerative Medicine Market Is Primarily Split Into

Cell Therapy

Tissue Engineering

Biomaterial

Other

On The Basis Of End Users/Application, This Report Covers

Dermatology

Cardiovascular

CNS

Orthopedic

Others

Regional Outlook of Regenerative Medicine Market report includes the following geographic areas such as: North America, Europe, China, Japan, Southeast Asia, India and ROW.

Influence of the Regenerative Medicine market report:

-Comprehensive assessment of all opportunities and risk in the Regenerative Medicine market.

Regenerative Medicine market recent innovations and major events.

-Detailed study of business strategies for growth of the Regenerative Medicine market-leading players.

-Conclusive study about the growth plot of Infrared Imaging market for forthcoming years.

-In-depth understanding of Regenerative Medicine market-particular drivers, constraints and major micro markets.

-Favourable impression inside vital technological and market latest trends striking the Regenerative Medicine market.

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Key Strategic Developments:The study also includes the key strategic developments of the market, comprising R&D, new product launch, M&A, agreements, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a global and regional scale.

Analytical Tools:The Global Regenerative Medicine Market Report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, SWOT analysis, feasibility study, and investment return analysis have been used to analyze the growth of the key players operating in the market.

Key Market Features:The report evaluated key market features, including revenue, price, capacity, capacity utilization rate, gross, production, production rate, consumption, import/export, supply/demand, cost, market share, CAGR, and gross margin. In addition, the study offers a comprehensive study of the key market dynamics and their latest trends, along with pertinent market segments and sub-segments.

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Regenerative Medicine Market 2021 Value with Status and Global Analysis DePuy Synthes, Osiris Therapeutics, Medtronic, Stryker, Acelity, ZimmerBiomet...

Rabbi Elie Abadie, M.D., senior rabbi, Jewish Council of the Emirates and Association of Gulf Jewish Communities.

Jonathan J. Bush, Jr., executive chair, Firefly Health.

Amy Abernethy, M.D., Ph.D., former principal deputy commissioner and acting chief information officer, U.S. Food and Drug Administration.

Deepak Chopra, M.D., founder, The Chopra Foundation and founder, Chopra Global.

Micah Aberson, executive vice president, Sanford Health.

George Church, Ph.D., founding core faculty and lead, Wyss Institute, Harvard University; professor of genetics, Harvard Medical School; professor of health sciences and technology, Harvard and Massachusetts Institute of Technology.

Gina Agiostratidou, Ph.D., program director, Type 1 diabetes program, The Leona M. and Harry B. Helmsley Charitable Trust.

Ellen Wright Clayton, M.D., J.D., Craig-Weaver Professor of pediatrics and professor of health policy, Center for Biomedical Ethics and Society, Vanderbilt University Medical Center and professor of law, Vanderbilt School of Law.

Rick Anderson, president and general manager, North America, DarioHealth.

Chelsea Clinton, D.Phil., M.P.H., vice chair, Clinton Foundation.

Stphane Bancel, CEO, Moderna.

Kelly L. Close, co-founder and chair of the board, The diaTribe Foundation.

Justin L. Barrett, Ph.D., president, Blueprint 1543, honorary professor of theology and the sciences, St. Andrews University, School of Divinity.

Francis S. Collins, M.D., Ph.D., director, U.S. National Institutes of Health.

Nir Barzilai, M.D., The Rennert Chair of Aging Research, professor of medicine and genetics and director, Nathan Shock Center of Excellence in the Biology of Aging, Albert Einstein College of Medicine.

Cindy Crawford, model and entrepreneur.

Marc Benioff, chair and CEO, Salesforce.

John F. Crowley, chair of the board and CEO, Amicus Therapeutics, Inc.

Paul Bloom, Ph.D., Brooks and Suzanne Ragen Professor of Psychology, Yale University.

Ray Dalio, founder, co-chair and co-chief information officer, Bridgewater Associates, New York Times best-selling author of "Principles: Life & Work."

Emma Bloomberg, founder and CEO, Murmuration.

Richard J. Davidson, Ph.D., professor of psychology and psychiatry, and founder and director of the Center for Healthy Minds, University of Wisconsin-Madison.

Albert Bourla, D.V.M., Ph.D., chair and CEO, Pfizer.

Grand Hospitaller, H.E. Dominique Prince de La Rochefoucauld-Montbel, Bailiff Grand Cross of Honour and Devotion in Obedience, The Sovereign Order of Malta.

Otis W. Brawley, M.D., Bloomberg Distinguished Professor of oncology and epidemiology, Johns Hopkins University.

Marilyn Glassberg, M.D., division chief of pulmonary medicine, critical care and sleep medicine; senior director of clinical research for strategy and growth, department of internal medicine, University of Arizona College of Medicine.

Dan Buettner, founder, Blue Zones.

Laurie H. Glimcher, M.D., president and CEO, Dana-Farber Cancer Institute, Richard and Susan Smith professor of medicine, Harvard Medical School, director, Dana-Farber/Harvard Cancer Center.

Ronald A. DePinho, M.D., Distinguished University Professor and past president, MD Anderson Cancer Center.

Dr. Jane Goodall, DBE, founder, the Jane Goodall Institute and U.N. Messenger of Peace.

Joseph M. DeVivo, president, hospital and health systems, Teladoc Health.

Scott Gottlieb, M.D., resident fellow, American Enterprise Institute and 23rd commissioner, U.S. Food and Drug Administration.

Spencer P. Eccles, co-founder and managing partner, The Cynosure Group.

Andre Goy, M.D., physician-in-chief of Oncology Services, Hackensack Meridian Health;chair, John Theurer Cancer Center; founding chair of Oncology, Hackensack Meridian School of Medicine.

Rev. Terrence P. Ehrman, C.S.C., Ph.D., visiting assistant teaching professor, department of theology, University of Notre Dame.

Kurt J. Griffin, M.D., Ph.D., Todd and Linda Broin Chair for Diabetes Research; director of clinical trials, The Sanford Project, Sanford Research; and associate professor, pediatric endocrinology, Sanford School of Medicine, University of South Dakota.

Michael E. Farkouh, M.D., Peter Munk Chair in Multinational Clinical Trials; director, Heart & Stroke/Richard Lewar Centre of Excellence; vice-chair, research and professor of medicine, department of medicine, University of Toronto.

Sanjay Gupta, M.D., award-winning chief medical correspondent, CNN and neurosurgeon.

Paul Farmer, M.D., Ph.D., Kolokotrones University professor and chair, department of global health and social medicine, Harvard Medical School; co-founder and chief strategist, Partners In Health.

Robert J. Hariri, M.D., Ph.D., founder, chair, and CEO, Celularity.

Anthony S. Fauci, M.D., director, National Institute of Allergy and Infectious Diseases, U.S. National Institutes of Health.

Katherine High, M.D., president, therapeutics, AskBio.

Judy Faulkner, founder and CEO, Epic.

Marc Hodosh, founder and co-host, "LIFE ITSELF."

David Feinberg, M.D., vice president, head of Google Health, Google.

Donald Hoffman, Ph.D., professor emeritus of cognitive sciences, University of California, Irvine.

Rev. Kevin T. FitzGerald, S.J., Ph.D., John A. Creighton University Professor and chair, department of medical humanities, Creighton University.

Jacquelyn Kulinski, M.D., director of the preventive cardiology program and associate professor of medicine, Medical College of Wisconsin.

Rene Fleming, soprano; arts and health advocate; artistic advisor, John F. Kennedy Center for the Performing Arts.

Samarth Kulkarni, Ph.D., CEO, CRISPR Therapeutics.

Robert C. Garrett, CEO, Hackensack Meridian Health.

Timothy A. Lash, president, West Health Policy Center.

Rebekah E. Gee, M.D., CEO, Louisiana State University Health Care Services; former secretary, Louisiana Department of Health.

William W. Li, M.D., president, medical director and CEO, The Angiogenesis Foundation.

Debra Houry, M.D., M.P.H., director, National Center for Injury Prevention and Control, U.S. Centers for Disease Control and Prevention.

Peter Libby, M.D., cardiovascular specialist, Brigham and Women's Hospital and Mallinckrodt Professor of medicine, Harvard Medical School.

Ryan Howard, founder and CEO, 100Plus.

Dan Liljenquist, senior vice president and chief strategy officer, Intermountain Healthcare.

Mark Hyman, M.D., head of strategy and innovation, Cleveland Clinic Center for Functional Medicine.

Shelley Lyford, president and CEO, Gary and Mary West Foundation.

Elder William K. Jackson, M.D., General Authority Seventy, The Church of Jesus Christ of Latter-day Saints.

Peter Marks, M.D., Ph.D., director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration.

Jo Ann Jenkins, CEO, AARP.

Brandon Marshall, NFL athlete, co-founder, Project 375 and founder, House of Athlete.

Henry Ji, Ph.D., chair, president and CEO, Sorrento Therapeutics.

Mark McClellan, M.D., Ph.D., director, Duke-Robert J. Margolis, M.D., Center for Health Policy, and the Robert J. Margolis, M.D., Professor of Business, Medicine, and Policy, Duke University.

Thupten Jinpa, Ph.D., president, Compassion Institute.

Gary Mendell, founder and CEO, Shatterproof.

Carl June, M.D., the Richard W. Vague Professor in Immunotherapy and director of the Center for Cellular Immunotherapies, Perelman School of Medicine; director of the Parker Institute for Cancer Immunotherapy, University of Pennsylvania.

Jamie Metzl, J.D., Ph.D., founder and chair, OneShared.World.

Cigall Kadoch, Ph.D., professor, Dana-Farber Cancer Institute and Harvard Medical School; founder, Foghorn Therapeutics.

Matthew Might, Ph.D., professor and director, Hugh Kaul Precision Medicine Institute, University of Alabama at Birmingham.

Dean Kamen, president, DEKA R&D, chair ARMI, and founder, FIRST.

Rosalind Picard, Sc.D., professor, MIT Media Lab; chief scientist and chair, Empatica.

Allen J. Karp, executive vice president, healthcare management and transformation, Horizon Blue Cross Blue Shield of New Jersey.

Renato Poletti, president, Science and Faith (STOQ) Foundation and president, Foundation for Heritage and Cultural and Artistic Activities of the Church.

Kerry Kennedy, president, Robert F. Kennedy Human Rights.

Cardinal Gianfranco Ravasi, president, Pontifical Council for Culture.

Stephen K. Klasko, M.D., president, Thomas Jefferson University, CEO, Jefferson Health.

Elder Dale G. Renlund, M.D., Quorum of the Twelve Apostles, The Church of Jesus Christ of Latter-day Saints.

Aaron J. Kowalski, Ph.D., president and CEO, JDRF International.

David C. Rhew, M.D., chief medical officer and vice president of healthcare, Worldwide Commercial Business, Microsoft.

Maria Millan, M.D., president and CEO, California Institute for Regenerative Medicine.

Walter Ricciardi, M.D., full professor in hygiene, Universit Cattolica del Sacro Cuore, Rome; president, Mission Board for Cancer of the European Commission; president, World Federation of Public Health Associations.

Princess Dina Mired, immediate past president, Union for International Cancer Control; patron of SIOP; honorary president of EORTC; special envoy for NCD's Vital Strategies; member of WHO Expert Group for the Elimination of Cervical Cancer.

Sheri L. Robb, Ph.D., professor, Indiana University School of Nursing.

William C. Mobley, M.D., Ph.D., associate dean for neurosciences initiatives, and interim director of the Sanford Institute for Empathy and Compassion, department of neurosciences, University of California San Diego.

Robert S. Rosenson, M.D., Director of Metabolism and Lipids, professor of medicine and cardiology, Icahn School of Medicine at Mount Sinai.

Dariush Mozaffarian, M.D., Dr.P.H., dean, Friedman School of Nutrition Science and Policy, Tufts University.

Frank J. Sasinowski, J.D., M.P.H., director, Hyman, Phelps & McNamara.

Michael Murray, Ph.D., president, Arthur Vining Davis Foundations.

Roy Schoenberg, M.D., M.P.H., president and co-CEO, Amwell.

Julien Musolino, Ph.D., associate professor, department of psychology, Rutgers University.

John Sculley, managing partner, Sculley Advisors.

David B. Nash, M.D., founding dean emeritus and the Raymond C. and Doris N. Grandon Professor of Health Policy, Jefferson College of Population Health.

Stephen Shaya, M.D., managing director, Akkad Holdings; executive servant leader, J&B Medical.

Timothy O'Connor, Ph.D., Mahlon Powell Professor of Philosophy, Indiana University.

Jerrell W. Shelton, chair, president and CEO, Cryoport.

Emmanuel "Manny" Ohonme, president and CEO, Samaritan's Feet International.

Andrew C. von Eschenbach, M.D., president, Samaritan Health Initiatives; former Commissioner U.S. Food and Drug Administration and 12th Director, National Cancer Institute.

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The Vatican's Pontifical Council for Culture and The Cura Foundation "Unite to Prevent" - PRNewswire

Particle sorting is a fundamental method in various fields of medical and biological research. However, existing sorting applications are not capable for high-throughput sorting of large-size (>100 micrometers) particles. Here, we present a novel on-chip sorting method using traveling vortices generated by on-demand microjet flows, which locally exceed laminar flow condition, allowing for high-throughput sorting (5 kilohertz) with a record-wide sorting area of 520 micrometers. Using an activation system based on fluorescence detection, the method successfully sorted 160-micrometer microbeads and purified fossil pollen (maximum dimension around 170 micrometers) from lake sediments. Radiocarbon dates of sorting-derived fossil pollen concentrates proved accurate, demonstrating the methods ability to enhance building chronologies for paleoenvironmental records from sedimentary archives. The method is capable to cover urgent needs for high-throughput large-particle sorting in genomics, metabolomics, and regenerative medicine and opens up new opportunities for the use of pollen and other microfossils in geochronology, paleoecology, and paleoclimatology.

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Breakthrough in purification of fossil pollen for dating of sediments by a new large-particle on-chip sorter - Science Advances

DUBLIN--(BUSINESS WIRE)--The "Growth Opportunities in Gene Therapies, Cancer, Disease Treatment & Monitoring, Surgical Applications and Cosmetics" report has been added to ResearchAndMarkets.com's offering.

This edition of the Life Science, Health & Wellness Technology Opportunity Engine (TOE) consists of insights across gene therapies, cancer, neurological, allergic and respiratory diseases treatment and monitoring. Further, novel drugs, drug delivery systems, surgical support applications, artificial intelligence-based imaging, as well as telehealth systems are discussed. Some innovations cover the upcoming innovations in cosmetics, acne treatment, food packing, and natural extract processing technologies

The Life Science, Health & Wellness TOE will feature disruptive technology advances in the global life sciences industry. The technologies and innovations profiled will encompass developments across genetic engineering, drug discovery and development, biomarkers, tissue engineering, synthetic biology, microbiome, disease management, as well as health and wellness among several other platforms.

The Health & Wellness cluster tracks developments in a myriad of areas including genetic engineering, regenerative medicine, drug discovery and development, nanomedicine, nutrition, cosmetic procedures, pain and disease management and therapies, drug delivery, personalized medicine, and smart healthcare.

Innovations in Life Sciences, Health & Wellness

For more information about this report visit https://www.researchandmarkets.com/r/2d6ood

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2021 Growth Opportunities in Gene Therapies, Cancer, Disease Treatment & Monitoring, Surgical Applications and Cosmetics - ResearchAndMarkets.com...

(Precision Vaccinations)

Florida-based Longeveron Inc. announced the completion of the Companys Phase I/II clinical study of the use of Lomecel-B to improve immune response to influenza vaccine in subjects with Aging Frailty.

Longeveron stated in a press release issued on April 15, 2021, 'it is anticipated that the top-line trial results will be announced in the 3rdquarter of 2021.'

Lomecel-B is an allogeneic, bone marrow-derived medicinal signaling cellproduct manufactured under current good manufacturing practicesby Longeveron.

The company says 'Lomecel-B has the potential to reduce inflammation associated with Aging Frailty and to promote an anti-inflammatory state by releasing anti-inflammatory molecules, which can balance the immune system and improve the function of B lymphocytes.

As B cells are responsible for antibody production in response to vaccines, Lomecel-B may boost antibody generation and immunity following vaccination in subjects with Aging Frailty.'

Completion of this clinical study to investigate Lomecel-B as a new therapeutic approach to boost immune response serves as an important initial step to meet the critical unmet medical need for those with Aging Frailty, who often respond poorly to vaccines, said Sean Leng, MD, Ph.D., Professor of Medicine, Molecular Microbiology and Immunology at Johns Hopkins University School of Medicine and Bloomberg School of Public Health and the studys principal investigator.

Aging Frailty is a life-threatening geriatric condition affecting approximately 15% of Americans over 65 or 8.1 million individuals. Aging Frailty patients are vulnerable to poor clinical outcomes compared to their age-matched peers despite sharing similar comorbidities and demographics. Therefore it is considered by some as an extreme form of unsuccessful aging.

Geoff Green, CEO of Longeveron. From the inception of Longeveron, we have focused our efforts on using a regenerative medicine approach to treat chronic, aging-related diseases and conditions, such as frailty and Alzheimers disease, with the goal of improving healthspan.

Miami-basedLongeveron is a clinical-stage biotechnology company developing cellular therapies for specific aging-related and life-threatening conditions.

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Infusion Improves Immune Response of Aging Frailty Following Flu Shot - PrecisionVaccinations

Roches gene therapy unit Spark Therapeutics has penned a $645 million-plus biobucks pact with Bayer-backed Senti Biosciences for new tech aimed at tweaking next-gen gene therapies.

Under the deal, Spark will maneuver Senti Bios gene circuit tech to drive the development of gene therapy 2.0. specifically directed toward specific cell types in the central nervous system, eye or liver.

Fierce 15 winner Senti Bios approach involves what it calls gene circuits, essentially tweaking a cells genetic code which can morph in order to address the severity of a disease, all the while limiting side effects.

Internally, the Californian biotech is applying its gene circuit tech specifically toward allogeneic chimeric antigen receptor natural killer cells, a growing area of interest in oncology.

Senti Bios lead assets out of this platform include SENTI-202 for acute myeloid leukemia, SENTI-301 for hepatocellular carcinoma and others for undisclosed solid tumor targets.

And its not just cancer: Senti Bio believes its on to something big, and it says it can also target a whole host of other areas and gene therapy delivery modalities such as immunology, neuroscience, cardiovascular disease, regenerative medicine and genetic diseases.

At the start of the year, it got off a major $105 million series B funding round, building on the initial $53 million it got off three years back. It also has the backing of some big names, with Leaps by Bayer and Amgens VC arm as well as Matrix Partners China, Mirae Asset Capital, Ridgeback Capital, Intel Capital, New Enterprise Associates, 8VC and Lux Capital all pitching in for its latest cash haul.

RELATED: Spark Therapeutics nabs CMO from new owner Roche

Now, it has a major pact with Spark/Roche that will see the biotech nab an undisclosed upfront payment and a mixture of biobucks, all of which could see Senti Bio bring home $645 million-plus.

For its part, Senti will be responsible for designing, building and testing cell type- and disease specific-synthetic promoters for use in certain CNS-, ocular- or liver-directed gene therapies.

Spark, meanwhile, holds an option to exclusively license a defined number of synthetic promoters emerging from the collab for use in developing gene therapy products in specified indications.

Should it hit go on that option, Spark will be responsible for conducting preclinical, clinical and sales work for any gene therapies that use Senti Bios licensed synthetic promoters.

We view gene circuits as a critical component of any advanced cell and gene therapy, regardless of therapeutic area or delivery modality, said Tim Lu, M.D., Ph.D., CEO of Senti Bio.

This collaboration with Spark Therapeutics aligns with our goal of enabling truly dynamic therapies that have the ability to discriminate between certain cell types, selectively express various payloads, and respond to diverse disease environments.

We are extremely impressed by the capabilities and know-how of Spark Therapeutics specifically in the area of gene therapy, and we look forward to bringing our mutual expertise together under this collaboration to harness the power of gene circuits to develop gene therapies that are clinically meaningful to patients.

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Senti Biosciences in $645M Spark pact to drive gene circuits into the fast lane - FierceBiotech

Researchers have genetically engineered a probiotic yeast to produce beta-carotene in the guts of laboratory mice. The advance demonstrates the utility of work the researchers have done to detail how a suite of genetic engineering tools can be used to modify the yeast.

"There are clear advantages to being able to engineer probiotics so that they produce the desired molecules right where they are needed," says Nathan Crook, corresponding author of the study and an assistant professor of chemical and biomolecular engineering at North Carolina State University. "You're not just delivering drugs or nutrients; you are effectively manufacturing the drugs or nutrients on site."

The study focused on a probiotic yeast called Saccharomyces boulardii. It is considered probiotic because it can survive and thrive in the gut, whereas most other yeast species either can't tolerate the heat or are broken down by stomach acid. It also can inhibit certain gut infections.

Previous research had shown that it was possible to modify S. boulardii to produce a specific protein in the mouse gut. And there are many well-established tools for genetically engineering baker's yeast, S. cerevisiae - which is used in a wide variety of biomanufacturing applications. Crook and his collaborators wanted to get a better understanding of which genetic engineering tools would work in S. boulardii.

Specifically, the researchers looked at two tools that are widely used for gene editing with the CRISPR system and dozens of tools that were developed specifically for modifying S. cerevisiae.

"We were a little surprised to learn that most of the S. cerevisiae tools worked really well in S. boulardii," Crook says. "Honestly, we were relieved because, while they are genetically similar, the differences between the two species are what make S. boulardii so interesting, from a therapeutic perspective."

Once they had established the viability of the toolkit, researchers chose to demonstrate its functionality modifying S. boulardii to produce beta-carotene. Their rationale was both prosaic and ambitious.

"On the one hand, beta-carotene is orange - so we could tell how well we were doing just by looking at the colonies of yeast on a petri dish: they literally changed color," Crook says. "On a more ambitious level, we knew that beta-carotene is a major provitamin A carotenoid, which means that it can be converted into vitamin A by the body - and we knew that vitamin A deficiency is a major public health problem in many parts of the world. So why not try to develop something that has the potential to be useful?"

Researchers tested the modified S. boulardii in a mouse model and found that the yeast cells successfully created beta-carotene in the guts of mice.

"This is a proof of concept, so there are a lot of outstanding questions," Crook says. "How much of this beta-carotene is getting absorbed by the mice? Are these biologically relevant amounts of beta-carotene? Would it work in humans? All of those are questions we'll have to address in future work. But we're excited to see what happens. And we're excited that these tools are now publicly available for use by others in the research community."

Reference:Durmusoglu D, AlAbri IS, Collins SP, et al. In situ biomanufacturing of small molecules in the mammalian gut by probiotic Saccharomyces boulardii. ACS Synth Biol. 2021. doi:10.1021/acssynbio.0c00562

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Probiotic Yeast Engineered To Produce Beta-Carotene - Technology Networks

This spring, the biotechnology company Oxitec plans to release genetically modified (GM) mosquitoes in the Florida Keys. Oxitec says its technology will combat dengue fever, a potentially life-threatening disease, and other mosquito-borne viruses such as Zika mainly transmitted by the Aedes aegypti mosquito.

While there have been more than 7,300 dengue cases reported in the United States between 2010 and 2020, a majority are contracted in Asia and the Caribbean, according to the US Centres for Disease Control and Prevention. In Florida, however, there were 41 travel-related cases in 2020, compared with 71 cases that were transmitted locally.

Native mosquitoes in Florida are increasingly resistant to the most common form of control insecticide and scientists say they need new and better techniques to control the insects and the diseases they carry. There arent any other tools that we have. Mosquito nets dont work. Vaccines are under development but need to be fully efficacious, says Michael Bonsall, a mathematical biologist at the University of Oxford, who is not affiliated with Oxitec but has collaborated with the company in the past, and who worked with the WHO to produce a GM mosquito-testing framework.

Bonsall and other scientists think a combination of approaches is essential to reducing the burden of diseases and that, maybe, newer ideas like GM mosquitoes should be added to the mix. Oxitecs mosquitoes, for instance, are genetically altered to pass what the company calls self-limiting genes to their offspring; when released GM males breed with wild female mosquitoes, the resulting generation does not survive into adulthood, reducing the overall population.

But Oxitec has been proposing to experimentally release GM mosquitos in the Keys since 2011, and the plan has long been met with suspicion among locals and debate among scientists. Some locals say they fear being guinea pigs. Critics say they are concerned about the possible effects GM mosquitoes could have on human health and the environment. In 2012, the Key West City Commissionobjected to Oxitecs plan; in a non-binding referendum four years later, residents of Key Haven where the mosquitoes would have been released rejected it, while residents in the surrounding county voted in support of the release. With the decision left up to the Florida Keys Mosquito Control District, officials approved the trial to be conducted elsewhere in the Keys.

According to Oxitec, the release was delayed due to a transfer of jurisdiction over the project from the U.S. Food and Drug Administration to the Environmental Protection Agency.

The company reapplied for approval to release a new version of the mosquitoes, called OX5034, in the Keys. In May, the EPA granted a two-year experimental use permit, which the agency can cancel at any time. State and local sign-off soon followed finally giving the project the greenlight.

Oxitecs OX5034 mosquitoes are the first GM mosquitoes approved for release in the US. The company has already conducted a trial with the OX5034 mosquitoes in Brazil and released more than a billion of a previous version, called OX513A, there and in other locations over the years including the Cayman Islands. The company says it is confident in the effectiveness and safety of the technology.

But some scientists want to hit pause on Oxitecs Florida trial, to find what they say is a fairer process in deciding to release the mosquitoes. Others want to see clearer proof that this technology is even necessary, claiming that the company has only released its most positive data with the public and has kept other key data, including whether the mosquitoes curb disease transmission, private. And if the release actually launches as planned, some Keys residents say they aim to interfere.

Critics also say that Oxitec failed to engage with local communities in Florida and get their consent to release the mosquitoes. Whats the most upsetting is that the very people that are going to be most impacted, both by the benefits or the risks of such a decision, have like the smallest voice in how these choices are made. I think thats a really big issue, says Natalie Kofler, a molecular biologist and bioethicist who founded Editing Nature, a platform that advocates for inclusive decision-making processes to steer the use of genetic technology. If Oxitec doesnt do this right, she adds, we could have a huge impact on delaying the use of other beneficial technologies like that in the future.

Oxitecs OX5034 mosquitoes are programmed to combat the transmission of mosquito-borne illnesses by suppressing local Aedes aegypti populations. Oxitec which is US-owned and based in the United Kingdom describes their mosquitoes as friendly because they will only release males, which, unlike females, do not bite humans or transmit disease.

Also read: Clever Approach: Scientists Create GM-Free Organisms Using Genetic Engineering

At Oxitecs laboratory in the UK, the company genetically engineers the mosquitoes, giving the insects the self-limiting gene that makes the females dependent on the antibiotic tetracycline. Without the drug, they will die. Eggs from these genetically-altered mosquitoes which will hatch both male and female insects will be shipped to the Keys. Mosquitoes require water to mature from an egg to an adult; when Oxitecs team adds water to the boxes the mosquitoes will be deployed in, both GM males and GM females will hatch. With no tetracycline present in the box, the GM females are expected to die in early larval stages.

The male mosquitoes will survive and carry the gene. When they leave the boxes, the insects will, hypothetically, fly away to mate with wild females to pass the gene to the next wild generation, according to Nathan Rose, head of regulatory affairs at Oxitec. Kevin Gorman, the companys chief development officer, says the local female mosquito population will be increasingly reduced which will also reduce the number of wild male mosquitoes in the treatment areas.

Gorman emphasised to Undark that the EPA and other regulators found no risk in using tetracycline in breeding their genetically-altered mosquitoes. But some scientists think the presence of this antibiotic in the environment does pose a risk. According to Jennifer Kuzma, co-founder and co-director of the Genetic Engineering and Society Centre at North Carolina State University, tetracyline is commonly used in Florida to prevent bacterial diseases in agriculture particularly in citrus groves and to treat bacteria in sewage plants.

The use of the antibiotic for these purposes may mean that it will remain in the environment, especially in water where the mosquitoes breed, which could allow Oxitecs female mosquitoes to survive. While the company does not plan to release the mosquitos near areas where the antibiotic is used, Kuzma says the EPAs risk assessment did not include testing of any standing water for tetracycline something, she adds, would have been easy enough to do for good due diligence.

Skeptics of Oxitecs GM mosquitoes include local residents, physicians, scientists and environmental activists. Many of these opponents say they arent anti-GMO, but disagree with how the approval process has been handled. One group has even kept a running list of what it sees as Oxitecs wrongdoings since it first began experimental releases. The list includes Oxitecs lack of disease monitoring in the countries where it has released mosquitoes; the unknown price of its technology; and complaints that the company has overstated the success of some of it its trials.

I cannot trust this company. I cannot trust this technology, says Mara Daly, a resident of Key Largo who says shes been following Oxitecs plans for nine years.

This is not a traditional pesticide, she adds. This is not a chemical that you can trace. This is something completely different, new emerging technology, and we need better regulation.

Phil Goodman, chairman of the Florida Keys Mosquito Control District (FKMCD), an independently-elected commission carrying out mosquito control within Monroe County, says that many of those who discredit Oxitecs evidence do not understand the technology. Theyre fear-mongering, he says.

They have very little credibility here in the Florida Keys as far as Im concerned, he adds.

But people like Daly and Barry Wray, executive director of the Florida Keys Environmental Coalition, disagree. We want to know its safe, says Wray, who notes that his group more generally supports GM technology. We dont have another Florida Keys ecosystem. We dont have another Florida Keys community. We have this one.

Daly, Wray, and others point to what they perceive as the FKMCDs disrespect for public opinion. They argue that the community wasnt given a chance to consent before the EPA approval. There was a 30-day public forum in September 2019 about Oxitecs technology application, with 31,174 comments opposing release and 56 in support. A statement emailed to Undark by Melissa Sullivan, an EPA spokesperson, noted that the agency considered these comments during the review, but critics think it happened too quickly to be of real use.

In June, Kofler and Kuzma wrote an opinion piece in The Boston Globe about the EPA approval, critiquing the agencys regulatory system and calling for a better process for evaluating new biotechnologies. The researchers expressed concern that the EPA did not convene an independent, external scientific advisory panel to review Oxitecs claims about its mosquito strategy and that the agency only publicly released its risk assessment after approving the technology. The American public, Kofler and Kuzma wrote, needs to be assured that these decisions are made free of conflicts of interest. The statement from the EPAs Sullivan noted that the agency conducted anextensive risk assessment based on the best available science.

Some critics also wanted there to be more public engagement. Kofler and Kuzma say they offered to provide their expertise, along with other outside experts, to the mosquito control district to allow more discussion about the GM mosquitoes with the Keys community. But Kofler says the district wasnt responsive. Oxitec itself launched webinars about their new product, but not until after the EPA approval. Here we are, like in the final hour, having these conversations that needed to be happening a year ago, says Kofler.

Without public trust and enthusiasm, it doesnt matter whether Oxitecs mosquito technique works, says Guy Reeves, a genetic researcher at the Max Planck Institute for Evolutionary Biology in Germany, who stresses that he doesnt think the companys approach is unsafe. If the population in Florida Keys becomes so sensitised to this issue that they can no longer cooperate with each other thats good for the mosquitoes, not good for the people, he adds.

Based on their first generation mosquito OX513A, Oxitec says it has shown that the approach reduces a targeted mosquito population in trials in both Brazil and the Cayman Islands. But theres no evidence that this new OX5034 mosquito release will actually be worth it for mosquito suppression, says Reeves. Oxitec also hasnt explained how their new mosquito will directly curb human diseases, such as dengue. Reducing disease transmission and burden should be measures of efficacy for this technology, says Kofler.

According to Gorman, independent disease suppression data has only been collected by municipalities in Brazil because thats where most of the companys trials have been released in larger scales. These municipalities have shown that Oxitec mosquitoes have reduced dengue cases in areas of release, Gorman says. In order for Oxitec to collect additional data, he adds, the company needs to release and test large areas over sustained periods of time. Gorman maintains that the company is not required to report formal health impact studies.

Reeves adds that Oxitec also hasnt explained what resources are needed to sustain this product, how long it could take to be effective, or the cost. When asked about the cost of the Florida Keys project, Oxitec responded to Undark by email: Oxitec is a pre-commercial, pre-profit company. We will not profit from this pilot project in Florida. We are paying for it ourselves.

Oxitec has released more than a billion of their OX513A mosquitoes over the past 10 years. According to independent scientists, some of those experiments did not go well.

For example, researchers at Yale University and collaborators from Brazil analysed Oxitecs 2015 release of OX513A in Brazil. The scientists confirmed that some offspring of the genetically modified mosquitoes which were supposed to die and not pass new genes to the wild population survived to adulthood and mated with their native counterparts. Between 10 and 60 percent of the native mosquitoes contained genes from Oxitec, according to the Yale study, which published in Nature in 2019. The papers authors concluded they do not know what impacts these mixed mosquitoes have on disease control or transmission, but added that their findings underscore the importance of monitoring the genetics of the insects.

Oxitec disagreed with the findings and responded on the journals website. Oxitec told Gizmodo that Yales study includes numerous false, speculative, and unsubstantiated claims and statements about Oxitecs mosquito technology. And when Kofler and three other scientists wrote about Oxitecs Brazil trial in The Conversation, Oxitec pushed to have the article retracted, says Kofler.

For this coming release, some Key Largo locals are willing to act on their anger. Daly, for instance, says that if the mosquitoes are deployed in her neighbourhood, shell try to put insecticide in any box she finds or send it to an expert to test even if it means getting in trouble with the federal authorities. I already have my arresting officer and she said shes gonna clean her handcuffs for me, she says. I dont care.

Ideally, Daly says, it wont have to come to that. She and other locals hope to stop Oxitec before the latest mosquitos are delivered. Daly says she has been busy organising protests like one that happened recently in Key Largo and giving out yard signs to residents who dont want their property used in the trial. Locals are pissed off. So I have been busy getting the press to cover the local opposition, Daly wrote in an email to Undark.

The first flying insect or animal that can actually use our human blood for a friggin trial for a product to come to market without my consent, Daly says.

Thats my blood, she adds. Thats my sons blood. Thats my dogs blood.

Taylor White is a freelance journalist based in Cape Cod, MA and a graduate of the Science, Health & Environmental Reporting Program at the NYU school of journalism. Her work has appeared in NOVA GBH, Dana-Farber Cancer Institute, the American Association for the Advancement of Science, GenomeWeb, Spectrum and Science Vs.

This article was originally published on Undark. Read the original article.

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In the US, Imminent Release of Genetically Modified Mosquitoes To Fight Dengue - The Wire Science

Over the past decade, the CRISPR-Cas9 gene editing system has revolutionized genetic engineering, allowing scientists to make targeted changes to organisms DNA. While the system could potentially be useful in treating a variety of diseases, CRISPR-Cas9 editing involves cutting DNA strands, leading to permanent changes to the cells genetic material.

Now, in a paper published online in Cell on April 9, researchers describe a new gene editing technology called CRISPRoff that allows researchers to control gene expression with high specificity while leaving the sequence of the DNA unchanged. Designed by Whitehead Institute Member Jonathan Weissman, University of California San Francisco assistant professor Luke Gilbert, Weissman lab postdoc James Nuez and collaborators, the method is stable enough to be inherited through hundreds of cell divisions, and is also fully reversible.

The big story here is we now have a simple tool that can silence the vast majority of genes, says Weissman, who is also a professor of biology at MIT and an investigator with the Howard Hughes Medical Institute. We can do this for multiple genes at the same time without any DNA damage, with great deal of homogeneity, and in a way that can be reversed. It's a great tool for controlling gene expression.

The project was partially funded by a 2017 grant from the Defense Advanced Research Projects Agency to create a reversible gene editor. Fast forward four years [from the initial grant], and CRISPRoff finally works as envisioned in a science fiction way, says co-senior author Gilbert. It's exciting to see it work so well in practice.

Because these methods alter the underlying DNA sequence, they are permanent. Plus, their reliance on in-house cellular repair mechanisms means it is hard to limit the outcome to a single desired change. As beautiful as CRISPR-Cas9 is, it hands off the repair to natural cellular processes, which are complex and multifaceted, Weissman says. It's very hard to control the outcomes.

Thats where the researchers saw an opportunity for a different kind of gene editor one that didnt alter the DNA sequences themselves, but changed the way they were read in the cell.

This sort of modification is what scientists call epigenetic genes may be silenced or activated based on chemical changes to the DNA strand. Problems with a cells epigenetics are responsible for many human diseases such as Fragile X syndrome and various cancers, and can be passed down through generations.

Epigenetic gene silencing often works through methylation the addition of chemical tags to to certain places in the DNA strand which causes the DNA to become inaccessible to RNA polymerase, the enzyme which reads the genetic information in the DNA sequence into messenger RNA transcripts, which can ultimately be the blueprints for proteins.

Weissman and collaborators had previously created two other epigenetic editors called CRISPRi and CRISPRa but both of these came with a caveat. In order for them to work in cells, the cells had to be continually expressing artificial proteins to maintain the changes.

With this new CRISPRoff technology, you can [express a protein briefly] to write a program that's remembered and carried out indefinitely by the cell, says Gilbert. It changes the game so now you're basically writing a change that is passed down through cell divisions in some ways we can learn to create a version 2.0 of CRISPR-Cas9 that is safer and just as effective, and can do all these other things as well.

Because the method does not alter the sequence of the DNA strand, the researchers can reverse the silencing effect using enzymes that remove methyl groups, a method they called CRISPRon.

As they tested CRISPRoff in different conditions, the researchers discovered a few interesting features of the new system. For one thing, they could target the method to the vast majority of genes in the human genome and it worked not just for the genes themselves, but also for other regions of DNA that control gene expression but do not code for proteins. That was a huge shock even for us, because we thought it was only going to be applicable for a subset of genes, says first author Nuez.

Also, surprisingly to the researchers, CRISPRoff was even able to silence genes that did not have large methylated regions called CpG islands, which had previously been thought necessary to any DNA methylation mechanism.

What was thought before this work was that the 30 percent of genes that do not have a CpG island were not controlled by DNA methylation, Gilbert says. But our work clearly shows that you don't require a CpG island to turn genes off by methylation. That, to me, was a major surprise.

The researchers chose a gene to silence in the stem cells, and then induced them to turn into nerve cells called neurons. When they looked for the same gene in the neurons, they discovered that it had remained silenced in 90 percent of the cells, revealing that cells retain a memory of epigenetic modifications made by the CRISPRoff system even as they change cell type.

They also selected one gene to use as an example of how CRISPRoff might be applied to therapeutics: the gene that codes for Tau protein, which is implicated in Alzheimers disease. After testing the method in neurons, they were able to show that using CRISPRoff could be used to turn Tau expression down, although not entirely off. What we showed is that this is a viable strategy for silencing Tau and preventing that protein from being expressed, Weissman says. The question is, then, how do you deliver this to an adult? And would it really be enough to impact Alzheimer's? Those are big open questions, especially the latter.

Even if CRISPRoff does not lead to Alzheimers therapies, there are many other conditions it could potentially be applied to. And while delivery to specific tissues remains a challenge for gene editing technologies such as CRISPRoff, we showed that you can deliver it transiently as a DNA or as an RNA, the same technology that's the basis of the Moderna and BioNTech coronavirus vaccine, Weissman says.

Weissman, Gilbert, and collaborators are enthusiastic about the potential of CRISPRoff for research as well. Since we now can sort of silence any part of the genome that we want, it's a great tool for exploring the function of the genome, Weissman says.

Plus, having a reliable system to alter a cells epigenetics could help researchers learn the mechanisms by which epigenetic modifications are passed down through cell divisions. I think our tool really allows us to begin to study the mechanism of heritability, especially epigenetic heritability, which is a huge question in the biomedical sciences, Nuez says.Reference:Nuez JK, Chen J, Pommier GC, et al. Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing. Cell. 2021;0(0). doi:10.1016/j.cell.2021.03.025

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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CRISPRoff: A New Addition to the CRISPR Toolbox - Technology Networks

When it comes to Alzheimers versus science, science is on the losing side.

Alzheimers is cruel in the most insidious way. The disorder creeps up in some aging brains, gradually eating away at their ability to think and reason, whittling down their grasp on memories and reality. As the worlds population ages, Alzheimers is rearing its ugly head at a shocking rate. And despite decades of research, we have no treatmentnot to mention a cure.

Too much of a downer? The National Institutes of Health (NIH) agrees. In one of the most ambitious projects in biology, the NIH is corralling Alzheimers and stem cell researchers to come together in the largest genome editing project ever conceived.

The idea is simple: decades of research have found certain genes that seem to increase the chance of Alzheimers and other dementias. The numbers range over hundreds. Figuring out how each connects or influences anotherif at alltakes years of research in individual labs. What if scientists unite, tap into a shared resource, and collectively solve the case of why Alzheimers occurs in the first place?

The initiatives secret weapon is induced pluripotent stem cells, or iPSCs. Similar to most stem cells, they have the ability to transform into anythinga cellular Genie, if you will. iPSCs are reborn from regular adult cells, such as skin cells. When transformed into a brain cell, however, they carry the original genes of their donor, meaning that they harbor the original persons genetic legacyfor example, his or her chance of developing Alzheimers in the first place. What if we introduce Alzheimers-related genes into these reborn stem cells, and watch how they behave?

By studying these iPSCs, we might be able to follow clues that lead to the genetic causes of Alzheimers and other dementiaspaving the road for gene therapies to nip them in the bud.

The iPSC Neurodegenerative Disease Initiative (iNDI) is set to do just that. The project aims to stimulate, accelerate, and support research that will lead to the development of improved treatments and preventions for these diseases, the NIH said. All resulting datasets will be openly shared online, for anyone to mine and interpret.

In plain language? Lets throw all of our new biotech superstarswith CRISPR at the forefrontinto a concerted effort against Alzheimers, to finally gain the upper hand. Its an Avengers, assemble moment towards one of our toughest foesone that seeks to destroy our own minds from within.

Alzheimers disease was first recognized in the early 1900s. Ever since, scientists have strived to find the cause that makes a brain waste away.

The most prominent idea today is the amyloid hypothesis. Imagine a horror movie inside a haunted house with ghosts that gradually intensify in their haunting. Thats the amyloid horrora protein that gradually but silently builds up inside a neuron, the house, eventually stripping it of its normal function and leading to the death of anything inside. Subsequent studies also found other toxic proteins that hang around outside the neuron house that gradually poison the molecular tenants within.

For decades scientists have thought that the best approach to beat these ghosts was an exorcismthat is, to get rid of these toxic proteins. Yet in trial after trial, they failed. The failure rate for Alzheimers treatmentso far, 100 percenthas led some to call treatment efforts a graveyard of dreams.

Its pretty obvious we need new ideas.

A few years ago, two hotshots strolled into town. One is CRISPR, the wunderkind genetic sharpshooter that can snip way, insert, or swap out a gene or two (or more). The other is iPSCs, induced pluripotent stem cells, which are reborn from adult cells through a chemical bath.

The two together can emulate Dementia 2.0 in a dish.

For example, using CRISPR, scientists can easily insert genes related to Alzheimers, or its protection, into an iPSCeither that from a healthy donor, or someone with a high risk of dementia, and observe what happens. A brain cell is like a humming metropolitan area, with proteins and other molecules whizzing around. Adding in a dose of pro-Alzheimers genes, for example, could block up traffic with gunk, leading scientists to figure out how those genes fit into the larger Alzheimers picture. For the movie buffs out there, its like adding into a cell a gene for Godzilla and another for King Kong. You know both could mess things up, but only by watching what happens in a cell can you know for sure.

Individual labs have tried the approach since iPSCs were invented, but theres a problem. Because iPSCs inherit the genetic baseline of a person, it makes it really difficult for scientists in different labs to evaluate whether a gene is causing Alzheimers, or if it was just a fluke because of the donors particular genetic makeup.

The new iNDI plan looks to standardize everything. Using CRISPR, theyll add in more than 100 genes linked to Alzheimers and related dementias into iPSCs from a wide variety of ethnically diverse healthy donors. The result is a huge genome engineering project, leading to an entire library of cloned cells that carry mutations that could lead to Alzheimers.

In other words, rather than studying cells from people with Alzheimers, lets try to give normal, healthy brain cells Alzheimers by injecting them with genes that could contribute to the disorder. If you view genes as software code, then its possible to insert code that potentially drives Alzheimers into those cells through gene editing. Execute the program, and youll be able to observe how the neurons behave.

The project comes in two phases. The first focuses on mass-engineering cells edited with CRISPR. The second is thoroughly analyzing these resulting cells: for example, their genetics, how their genes activate, what sorts of proteins they carry, how those proteins interact, and so on.

By engineering disease-causing mutations in a set of well-characterized, genetically diverse iPSCs, the project is designed to ensure reproducibility of data across laboratories and to explore the effect of natural variation in dementia, said Dr. Bill Skarnes, director of cellular engineering at the Jackson Laboratory, and a leader of the project.

iNDI is the kind of initiative thats only possible with our recent biotech boost. Engineering hundreds of cells related to Alzheimersand to share with scientists globallywas a pipe dream just two decades ago.

To be clear, the project doesnt just generate individual cells. It uses CRISPR to make cell lines, or entire lineages of cells with the Alzheimers gene that can pass on to the next generation. And thats their power: they can be shared with labs around the world, to further hone in on genes that could make the largest impact on the disorder. Phase two of iNDI is even more powerful, in that it digs into the inner workings of these cells to generate a cheat codea sheet of how their genes and proteins behave.

Together, the project does the hard work of building a universe of Alzheimers-related cells, each outfitted with a gene that could make an impact on dementia. These types of integrative analyses are likely to lead to interesting and actionable discoveries that no one approach would be able to learn in isolation, the authors wrote. It provides the best chance at truly understanding Alzheimers and related diseases, and promising treatment possibilities.

Image Credit: Gerd Altmann from Pixabay

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A Massive New Gene Editing Project Is Out to Crush Alzheimer's - Singularity Hub

The human genome has long been a difficult book to read. Modern technological advances have recently opened doors for researchers to begin asking a big question: What parts of our DNA sequences might influence disease? Mary Lauren Benton, Ph.D., recently joined the Baylor Engineering and Computer Science faculty as an assistant professor of bioinformatics, and she is working to answer that question.

Mary Lauren Benton, Ph.D.

If you think of the genome like an instruction manual, Im interested in the grammar, Benton said. Im interested in understanding how short DNA sequences turn genes on and off in different cells and allow for many different outcomes. If we know how a particular sequence influences risk of heart disease, for example, we can use that information to help us guide clinical decisions, whether thats applying different treatments, prescribing different medications or scheduling more preventative care. All of these things can help clinicians to better prioritize and care for patients.

Benton uses computer modeling to look through large data sets of genetic information. Bioinformatics allows for processing of these large data in ways not possible previously, giving room for biological researchers to find patterns and solutions using methods and tools from computer science.

I think of bioinformatics as the intersection of computer science and biology, Benton said. I take tools and methods from computer science, and I apply them to solve fundamental biological questions. We have a lot of really big data sets in biology. The human genome is 3 billion base pairs long, which we cant analyze by hand. The tools from computer science and statistics give us a way to ask questions that we wouldnt be able to otherwise. They open the doors to analyses that would have been impossible even 10 or 20 years ago.

Benton most recently authored The Influence of Evolutionary History on Human Health and Disease, which was published in the Nature Reviews Genetic Journal and takes a look at the evolutionary origins of disease. Being diagnosed with a disease or health problem may feel like a present problem; however, Benton explained that looking at the foundations of a disease is important to understanding how to move forward with treatment.

The foundations and the systems that are involved in disease have really deep evolutionary origins, she said. Cancer might be something that youre diagnosed with today, but the foundation of cancer can be traced back to the idea that we have cells that are able to grow and divide, which also provides the opportunity for tumors to grow.

Benton explained that its important to consider the history of the disease and the systems involved alongside any variants or environmental factors that help to cause the disease. A holistic understanding of disease can influence how patients are treated as well as provide information about how their diseases came to be.

Its not enough to understand whats happening in a person right now or in the last five years, Benton said. Understanding the million-year history of how people got here is equally important to make advances in personalizing medicine, especially genomic medicine. Having that long lens is something that is often lost in the day-to-day operations of a doctors office.

Benton is excited to be evaluating the way that researchers think about decoding genetic information. While a common approach is to think of genes as being able to be turned off or on with a simple switch, that may not be the most accurate approach.

We study these sets of genetic switches and how they turn genes on and off at the right times. Often, we think about these switches working one-at-a-time; the gene is either on or its off, Benton said. But it is much more complicated than that. There are often multiple switches that act more like a dashboard of knobs and dials that all work together to properly tune the output of the genome.

Bentons research is moving toward the development of new models and ways of thinking about how known individual elements are combined and factored into this much larger, more accurate dashboard. Differences based on demographic histories, environmental variables and evolutionary processes all influence the risk of disease in different ways. A better understanding of genomes and how genetic variants relate to disease has major implications for precision medicine.

Its really vital for precision medicine to take into account the full diversity of the human experience. We cant focus on one particular kind of person or one population. People of European ancestry are over-represented in genetic studies, Benton said. Improving diversity and representation in our genomic studies is vital to understanding how the genome relates to disease and to learning how to appropriately treat all of the patients that might walk through the doors of a clinic.

Precision medicine, in some ways, seems futuristic and far-off. But, in other ways, precision medicine is already being used to protect at-risk individuals from diseases like cancer. While widespread precision medicine may not be seen for a long time, research like Bentons plays a role in better understanding disease risk broadly and providing context for clinical solutions moving forward.

Precision medicine is both happening right now and is something that well probably always be working toward, Benton said. There are things that we understand right now about specific genetic variants that might predispose you to a certain kind of breast cancer, for example. We already have diseases that we can test for or treat differently based on someones genotype. But, because the genome is such a complicated thing, walking into the clinic and handing your DNA sequence to the doctor, who would then read it and prescribe the right treatments on the spot, is a goal that well always be working toward. Still, I expect well see big changes in the next five to 10 years given the current rate of progress.

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Grammar of the Genome: Reading the Influence of DNA on Disease - Baylor University

The information and biotechnology revolutions have changed our world and will heavily inform the future of society. Whoever controls these technologies controls the future, and whoever controls their standardization controls the technologies. China understands this well. For two decades, it has been working to take over international standardization rulemaking bodies to serve the goals advanced in Made in China 2025 that is, to dominate world manufacturing and then transition to become the center of the worlds technological innovation

The dangers to the United States are already present, and in forms that are not obvious. These include, first, direct-to-consumer genetic testing. China may be using such testing to gain genetic information that permits the identification and tracking of Americans, including U.S. military and intelligence community personnel or their relatives. Second, health monitoring apps are able to provide geolocation data to Chinese entities, which means to the Chinese Communist Party (CCP) and its security services. This provides location data that is valuable on its own and might be compared with data from other sources to reveal key information about Americans. Third, the CCP, in cooperation with Chinese industrial entities on international bodies, are developing and setting international standards for emerging technologies. Chinas influence has grown over the past two decades, and Beijing now possesses leadership roles in standards-drafting technical committees, which means it could shape outcomes to its benefit.

China has formulated a four-step strategy to seek dominance in this area: plan, track, participate and take over. Beijing has boasted that it completed the first three steps and is on the last, which is to develop indigenous standards and to lead international standardization. This means China may be replacing international standards with its own standards, in order to control technologies and the market. In 2017, China revised its standardization law, almost 30 years after its adoption in 1989. It also set up the Standardization Administration of China to implement its strategy in the early 2000s. Chinas standardization strategy also has been incorporated into the Belt and Road Initiative so that, as countries are weaved into this network, they adopt Chinas standards.

Beijing essentially has had the three primary standard-setting international organizations the International Organization for Standardization (ISO), the International Telecommunication Union (ITU) and the International Electrotechnical Commission (IEC) under its influence. Two Chinese government officials currently serve as president of ITU and IEC, and placed Chinas proxy as the head of the ISO after the organization was led by a Chinese official for many years. Meanwhile, Beijing has taken leadership or other influential positions in the International Accreditation Forum (IAF), United Nations Industrial Development Organization (UNIDO), International Civil Aviation Organization (ICAO), American Society for Quality (ASQ) and perhaps others.

Chinas strategy to determine the worlds standards appears to be working. In 2019 alone, China submitted 830 standards proposals to the ITU. According to Zhang Xiaogang, former president of the ISO, China planned to initiate 395 international standards by 2020 but, in actuality, it set 495. Zhang claims that China has made the greatest contribution in the field of international standardization in the past five years. Indeed, China has dominated 5G standard-setting, for example, in the 3rd Generation Partnership Project (3GPP), an organization to develop mobile broadband standards, and 90 percent of standard proposals in the 5G super uplink field is done by China Telecom.

Unfortunately, Western countries fail to see the importance of Chinas strategic move. Zhang states, Whoever leads in standard-setting will be the leader of the technology and the controller of the market. Chinas dominance in 5G standards-setting enables it to avoid the Wests sanctions against its tech giants such as Huawei, continue to expand globally, and to dominate the market. This could be a paramount communication-security problem for the U.S.

Of particular importance is Chinas standardization strategy as identified in China Standards 2035 on international bodies engaged in developing and setting standards for select emerging technologies. These include advanced communication technologies and cloud computing and cloud services. The United States and its allies must ensure that international standards for emerging technologies are not being designed to promote the interests of China. If China is successful, it would lead to the exclusion of other participants; China would be the architect, builder and maintainer of the 21st centurys information technology infrastructure.

Washington must take steps to bolster U.S. public- and private-sector participation in international standards-setting bodies. The U.S. must underscore the dangers of interacting with Chinese firms. The government, particularly the Department of Homeland Security, has called attention to this, but there is little recognition that U.S. firms understand the risks associated. There must be greater awareness of smartphone apps, which should be graded on user privacy and their security from Chinese penetration. And U.S. entities that permit Chinese entities to exploit data should be subject to possible legal action.

A key question of the 21st century is a holdover from the previous century: Which state will control scientific knowledge and its standards? The answer is not yet clear. Thankfully, in the 20th century, the U.S. surpassed Nazi Germany to dominate physics and related sciences and industries, and generated the centurys most fearsome weapon the atomic bomb and its progeny, the hydrogen bomb.

If these revolutionary advances occur within the framework of the U.S.-led liberal international order, we are assured improvements to the well-being of people. But if China leads the scientific and technological revolution, these advances will serve the CCPs inherently malign interests. China will aggressively seize opportunities, particularly in nascent areas where standards are not developed including biotechnology, genetic engineering, energy production and distribution technologies, aerospace, 5G and artificial intelligence.

If China has its way in standards-setting, the communist regime will control these critical technologies and the global supply chain. That means it would dominate the future of the free worlds economy, media and politics. The United States no longer can afford complacency.

Bradley A. Thayer is the co-author of How China Sees the World: Han-Centrism and the Balance of Power in International Politics.

Lianchao Han is vice president of Citizen Power Initiatives for China. After the Tiananmen Square massacre in 1989, he was one of the founders of the Independent Federation of Chinese Students and Scholars. He worked in the U.S. Senate for 12 years, as legislative counsel and policy director for three senators.

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We cannot let China set the standards for 21st century technologies | TheHill - The Hill

This spring, the biotechnology company Oxitec plans to release genetically modified (GM) mosquitoes in the Florida Keys. Oxitec says its technology will combat dengue fever, a potentially life-threatening disease, and other mosquito-borne virusessuch as Zika mainly transmitted by the Aedes aegypti mosquito.

While there have been more than 7,300 dengue cases reported in the United States between 2010 and 2020, a majority are contracted in Asia and the Caribbean, according to the U.S. Centers for Disease Control and Prevention. In Florida, however, there were 41 travel-related cases in 2020, compared with 71 cases that were transmitted locally.

Native mosquitoes in Florida are increasingly resistant to the most common form of control insecticideand scientists say they need new and better techniques to control the insects and the diseases they carry. There arent any other tools that we have. Mosquito nets dont work. Vaccines are under development but need to be fully efficacious, says Michael Bonsall, a mathematical biologist at the University of Oxford, who is not affiliated with Oxitec but has collaborated with the company in the past, and who worked with the World Health Organization to produce a GM mosquito-testing framework.

Bonsall and other scientists think a combination of approaches is essential to reducing the burden of diseasesand that, maybe, newer ideas like GM mosquitoes should be added to the mix. Oxitecs mosquitoes, for instance, are genetically altered to pass what the company calls self-limiting genes to their offspring; when released GM males breed with wild female mosquitoes, the resulting generation does not survive into adulthood, reducing the overall population.

But Oxitec has been proposing to experimentally release GM mosquitos in the Keys since 2011, and the plan has long been met with suspicion among locals and debate among scientists. Some locals say they fear being guinea pigs. Critics say they are concerned about the possible effects GM mosquitoes could have on human health and the environment. In 2012, the Key West City Commissionobjected to Oxitecs plan; in a non-binding referendum four years later, residents of Key Havenwhere the mosquitoes would have been releasedrejected it, while residents in the surrounding county voted in support of the release. With the decision left up to the Florida Keys Mosquito Control District, officials approved the trial to be conducted elsewhere in the Keys.

According to Oxitec, the release was delayed due to a transfer of jurisdiction over the project from the U.S. Food and Drug Administration to the Environmental Protection Agency.

The company reapplied for approval to release a new version of the mosquitoes, called OX5034, in the Keys. In May, the EPA granted a two-year experimental use permit, which the agency can cancel at any time. State and local sign-off soon followedfinally giving the project the greenlight.

Oxitecs OX5034 mosquitoes are the first GM mosquitoes approved for release in the US. The company has already conducted a trial with the OX5034 mosquitoes in Brazil and released more than a billion of a previous version, called OX513A, there and in other locations over the yearsincluding the Cayman Islands. The company says it is confident in the effectiveness and safety of the technology.

But some scientists want to hit pause on Oxitecs Florida trial, to find what they say is a fairer process in deciding to release the mosquitoes. Others want to see clearer proof that this technology is even necessary, claiming that the company has only released its most positive data with the public and has kept other key data, including whether the mosquitoes curb disease transmission, private. And if the release actually launches as planned, some Keys residents say they aim to interfere.

Critics also say that Oxitec failed to engage with local communities in Florida and get their consent to release the mosquitoes. Whats the most upsetting is that the very people that are going to be most impacted, both by the benefits or the risks of such a decision, have like the smallest voice in how these choices are made. I think thats a really big issue, says Natalie Kofler, a molecular biologist and bioethicist who founded Editing Nature, a platform that advocates for inclusive decision-making processes to steer the use of genetic technology. If Oxitec doesnt do this right, she adds, we could have huge impact on delaying the use of other beneficial technologies like that in the future.

Oxitecs OX5034 mosquitoes are programmed to combat the transmission of mosquito-borne illnesses by suppressing local Aedes aegypti populations. Oxitecwhich is US-owned and based in the United Kingdomdescribes their mosquitoes as friendly because they will only release males, which, unlike females, do not bite humans or transmit disease.

At Oxitecs laboratory in the UK, the company genetically engineers the mosquitoes, giving the insects the self-limiting gene that makes the females dependent on the antibiotic tetracycline. Without the drug, they will die. Eggs from these genetically-altered mosquitoeswhich will hatch both male and female insectswill be shipped to the Keys. Mosquitoes require water to mature from an egg to an adult; when Oxitecs team adds water to the boxes the mosquitoes will be deployed in, both GM males and GM females will hatch. With no tetracycline present in the box, the GM females are expected to die in early larval stages.

The male mosquitoes will survive and carry the gene. When they leave the boxes, the insects will, hypothetically, fly away to mate with wild females to pass the gene to the next wild generation, according to Nathan Rose, head of regulatory affairs at Oxitec. Kevin Gorman, the companys chief development officer, says the local female mosquito population will be increasingly reducedwhich will also reduce the number of wild male mosquitoes in the treatment areas.

Gorman emphasized to Undark that the EPA and other regulators found no risk in using tetracycline in breeding their genetically-altered mosquitoes. But some scientists think the presence of this antibiotic in the environment does pose a risk. According to Jennifer Kuzma, co-founder and co-director of the Genetic Engineering and Society Center at North Carolina State University, tetracyline is commonly used in Florida to prevent bacterial diseases in agricultureparticularly in citrus grovesand to treat bacteria in sewage plants. The use of the antibiotic for these purposes may mean that it will remain in the environment, especially in water where the mosquitoes breed, which could allow Oxitecs female mosquitoes to survive. While the company does not plan to release the mosquitos near areas where the antibiotic is used, Kuzma says the EPAs risk assessment did not include testing of any standing water for tetracyclinesomething, she adds, would have been easy enough to do for good due diligence.

Skeptics of Oxitecs GM mosquitoes include local residents, physicians, scientists, and environmental activists. Many of these opponents say they arent anti-GMO, but disagree with how the approval process has been handled. One group has even kept a running list of what it sees as Oxitecs wrongdoings since it first began experimental releases. The list includes Oxitecs lack of disease monitoring in the countries where it has released mosquitoes; the unknown price of its technology; and complaints that the company has overstated the success of some of it its trials.

I cannot trust this company. I cannot trust this technology, says Mara Daly, a resident of Key Largo who says shes been following Oxitecs plans for nine years.

This is not a traditional pesticide, she adds. This is not a chemical that you can trace. This is something completely different, new emerging technology and we need better regulation.

Phil Goodman, chairman of the Florida Keys Mosquito Control District (FKMCD), an independently-elected commission carrying out mosquito control within Monroe County, says that many of those who discredit Oxitecs evidence do not understand the technology. Theyre fearmongering, he says.

They have very little credibility here in the Florida Keys as far as Im concerned, he adds.

But people like Daly and Barry Wray, executive director of the Florida Keys Environmental Coalition, disagree. We want to know its safe, says Wray, who notes that his group more generally supports GM technology. We dont have another Florida Keys ecosystem. We dont have another Florida Keys community. We have this one.

Daly, Wray, and others point to what they perceive as the FKMCDs disrespect for public opinion. They argue that the community wasnt given a chance to consent before the EPA approval. There was a 30-day public forum in September 2019 about Oxitecs technology application, with 31,174 comments opposing release and 56 in support. A statement emailed to Undark by Melissa Sullivan, an EPA spokesperson, noted that the agency considered these comments during the review, but critics think it happened too quickly to be of real use.

In June, Kofler and Kuzma wrote an opinion piece in The Boston Globe about the EPA approval, critiquing the agencys regulatory system and calling for a better process for evaluating new biotechnologies. The researchers expressed concern that the EPA did not convene an independent, external scientific advisory panel to review Oxitecs claims about its mosquito strategy and that the agency only publicly released its risk assessment after approving the technology. The American public, Kofler and Kuzma wrote, needs to be assured that these decisions are made free of conflicts of interest. The statement from the EPAs Sullivan noted that the agency conducted anextensive risk assessmentbased on the best available science.

Some critics also wanted there to be more public engagement. Kofler and Kuzma say they offered to provide their expertise, along with other outside experts, to the mosquito control district to allow more discussion about the GM mosquitoes with the Keys community. But Kofler says the district wasnt responsive. Oxitec itself launched webinars about their new product, but not until after the EPA approval. Here we are, like in the final hour, having these conversations that needed to be happening a year ago, says Kofler.

Without public trust and enthusiasm, it doesnt matter whether Oxitecs mosquito technique works, says Guy Reeves, a genetic researcher at the Max Planck Institute for Evolutionary Biology in Germany, who stresses that he doesnt think the companys approach is unsafe. If the population in Florida Keys becomes so sensitized to this issuethat they can no longer cooperate with each otherthats good for the mosquitoes, not good for the people, he adds.

Based on their first generation mosquito OX513A, Oxitec says it has shown that the approach reduces a targeted mosquito population in trials in both Brazil and the Cayman Islands. But theres no evidence that this new OX5034 mosquito release will actually be worth it for mosquito suppression, says Reeves. Oxitec also hasnt explained how their new mosquito will directly curb human diseases, such as dengue. Reducing disease transmission and burden should be measures of efficacy for this technology, says Kofler.

According to Gorman, independent disease suppression data has only been collected by municipalities in Brazil because thats where most of the companys trials have been released in larger scales. These municipalities have shown that Oxitec mosquitoes have reduced dengue cases in areas of release, Gorman says. In order for Oxitec to collect additional data, he adds, the company needs to release and test large areas over sustained periods of time. Gorman maintains that the company is not required to report formal health impact studies.

Reeves adds that Oxitec also hasnt explained what resources are needed to sustain this product, how long it could take to be effective, or the cost. When asked about the cost of the Florida Keys project, Oxitec responded to Undark by email: Oxitec is a pre-commercial, pre-profit company. We will not profit from this pilot project in Florida.We are paying for it ourselves.

Oxitec has released more than a billion of their OX513A mosquitoes over the past 10 years. According to independent scientists, some of those experiments did not go well.

For example, researchers at Yale University and collaborators from Brazil analyzed Oxitecs 2015 release of OX513A in Brazil. The scientists confirmed that some offspring of the genetically modified mosquitoeswhich were supposed to die and not pass new genes to the wild populationsurvived to adulthood and mated with their native counterparts. Between 10 and 60 percent of the native mosquitoes contained genes from Oxitec, according to the Yale study, which published in Nature in 2019. The papers authors concluded they do not know what impacts these mixed mosquitoes have on disease control or transmission, but added that their findings underscore the importance of monitoring the genetics of the insects.

Oxitec disagreed with the findings and responded on the journals website. Oxitec told Gizmodo that Yales study includes numerous false, speculative, and unsubstantiated claims and statements about Oxitecs mosquito technology. And when Kofler and three other scientists wrote about Oxitecs Brazil trial in The Conversation, Oxitec pushed to have the article retracted, says Kofler.

For this coming release, some Key Largo locals are willing to act on their anger. Daly, for instance, says that if the mosquitoes are deployed in her neighborhood, shell try to put insecticide in any box she finds or send it to an expert to testeven if it means getting in trouble with the federal authorities. I already have my arresting officer and she said shes gonna clean her handcuffs for me, she says. I dont care.

Ideally, Daly says, it wont have to come to that. She and other locals hope to stop Oxitec before the latest mosquitos are delivered. Daly says she has been busy organizing protests like one that happened recently in Key Largoand giving out yard signs to residents who dont want their property used in the trial. Locals are pissed off. So I have been busy getting the press to cover the local opposition, Daly wrote in an email to Undark.

The first flying insect or animal that can actually use our human blood for a friggin trial for a product to come to market without my consent, Daly says.

Thats my blood, she adds. Thats my sons blood. Thats my dogs blood.

This article was originally published on Undark. Read the original article.

Image Credit: Frauke Feind from Pixabay

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GAITHERSBURG, Md., April 14, 2021 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), a biotechnology company developing next-generation vaccines for serious infectious diseases, today announced its participation in a newly expanded investigator-initiated Phase 2 clinical trial called Comparing COVID-19 Vaccine Schedule Combinations Stage 2(Com-COV2), to be conducted by the University of Oxford and supported by the UK Vaccines Taskforce. Novavax' recombinant protein vaccine candidate, NVX-CoV2373, is one of four COVID-19 vaccines that will be studied to evaluate the potential for combined regimens that mix vaccines from different manufacturers to achieve immune protection against COVID-19.

"Novavax' addition to this important study reflects the urgency of finding innovative ways to protect as many people as possible in a dynamic pandemic landscape," said Filip Dubovsky, M.D., Executive Vice President, Chief Medical Officer, Novavax. "The potential utility of pooling public health resources, including all available vaccines, could help us get ahead of an evolving virus."

Com-COV2 will include 1050 adults 50 years of age or older who received their first vaccination during the prior 8-12 weeks. Volunteer study participants will receive one of four different vaccines as a second dose, 350 of whom will be administered NVX-CoV2373. The research will compare the immune system responses from those who receive a heterologous regimen to those who receive a homologous regimen.

"The focus of these studies is to explore whether multiple COVID-19 vaccines can be used more flexibly, with different vaccines being used for the first and second doses," said Matthew Snape, Associate Professor in Paediatrics and Vaccinology at the University of Oxford, and Chief Investigator on the trial. "If we can show that these mixed schedules generate an immune response that is as good as the standard schedules, this could potentially allow more people to complete their COVID-19 immunization course more rapidly."

Under the protocol, which is a designed as a non-inferiority study, participants will be followed for reactogenicity (safety) and immune responses. The UK Medicines and Healthcare products Regulatory Agency (MHRA) and Joint Committee on Vaccination and Immunisation (JCVI) will formally assess the safety and efficacy of any new vaccination regimen before it is made available to the public.

About NVX-CoV2373NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease. NVX-CoV2373 was created using Novavax' recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is adjuvanted with Novavax' patented saponin-based Matrix-M to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19. In preclinical studies, NVX-CoV2373 induced antibodies that blocked the binding of spike protein to cellular receptors and provided protection from infection and disease. It was generally well-tolerated and elicited robust antibody response in Phase 1/2 clinical testing.

NVX-CoV2373 is being evaluated in two pivotal Phase 3 trials, a trial in the U.K that demonstrated efficacy of 96.4% against the original virus strain and 89.7% overall, and the PREVENT-19 trial in the U.S. and Mexico that began in December 2020. It is also being tested in two ongoing Phase 2 studies that began in August 2020: A Phase 2b trial in South Africa that demonstrated 48.6% efficacy against a newly emerging escape variant, and a Phase 1/2 continuation in the U.S. and Australia.

NVX-CoV2373 is stored and stable at 2- 8C, allowing the use of existing vaccine supply chain channels for its distribution. It is packaged in a ready-to-use liquid formulation in 10-dose vials.

About Matrix-MNovavax' patented saponin-based Matrix-M adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.

About NovavaxNovavax, Inc.(Nasdaq: NVAX) is a biotechnology company that promotes improved health globally through the discovery, development and commercialization of innovative vaccines to prevent serious infectious diseases. The company's proprietary recombinant technology platform combines the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles designed to address urgent global health needs. Novavaxis conducting late-stage clinical trials for NVX-CoV2373, its vaccine candidate against SARS-CoV-2, the virus that causes COVID-19. NanoFlu, its quadrivalent influenza nanoparticle vaccine, met all primary objectives in its pivotal Phase 3 clinical trial in older adults and will be advanced for regulatory submission. Both vaccine candidates incorporate Novavax' proprietary saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

For more information, visit http://www.novavax.com and connect with us on Twitter and LinkedIn.

Novavax Forward Looking StatementsStatements herein relating to the future of Novavax and the ongoing development of its vaccine and adjuvant products are forward-looking statements. Novavax cautions that these forward-looking statements are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include those identified under the heading "Risk Factors" in the Novavax Annual Report on Form 10-K for the year ended December 31, 2020, as filed with the Securities and Exchange Commission (SEC). We caution investors not to place considerable reliance on forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

Contacts:

InvestorsNovavax, Inc.Erika Schultz | 240-268-2022ir@novavax.com

Solebury TroutJennifer Porcelli | 646-378-2962jporcelli@soleburytrout.com

Novavax MediaAmy Speak | 617-420-2461Laura Keenan | 410-419-5755media@novavax.com

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Novavax to Participate in University of Oxford Com-COV2 Study Comparing Mixed COVID-19 Vaccine Combinations - BioSpace

Washington State-based AmunBio and Chicago-based NorthShore University HealthSystems Research Institute have agreed to collaborate in the development and the commercialization of an innovative technology platform of novel immunotherapeutic oncolytic viruses. AmunBio has an exclusive option to license this technology.

The proprietary platform technology has the potential to generate a strong pipeline of patient-centric next-generation immunotherapeutic oncolytic viruses.

The conceptOncolytic viruses are a form of immunotherapy that uses native or reprogrammed viruses to infect and selectively kill cancerous cells. The concept of using viruses in killing cancer is, however, not new. At the turn of the nineteenth century, when the existence of viruses was first recognized, there has been considerable interest in using viruses as possible agents of tumor destruction.

The use of viruses in cancer treatment was not the result of true understanding and discerning theory of a possible therapy, but rather, was based on the observation that, in some cases, patients diagnosed with cancer or hematological malignancies who contracted an infectious disease went into brief periods of clinical remission

Early case reports emphasized regression of cancers during naturally acquired virus infections, providing the initial basis for clinical trials where body fluids containing human or animal viruses were used to transmit infections to cancer patients.

In one case, in a patient diagnosed with leukemia, it was well recognized that the contraction of influenza produced beneficial effects. And while doctors were not able to report cases where an accompanying infectious disease led to a complete cure of leukemia, they believed that a treatment based upon the causal infection would, potentially, provide an alternative to the hopelessness of the ordinary treatment of leukemia.'[1]

And while in this approach of infecting cancer patients the immune system arrested the impact of the viruses in most cases, and the viruses failed to impact tumor growth, when, in immunosuppressed patients, infection persisted, tumors regressed. However, morbidity as a result of the infection of normal tissues was unacceptably high, ending attempts to develop novel virus-based treatment options.[2]

In the 1950s and 1960s, with the advent of rodent models and new methods for virus propagation, researchers attempted to develop viruses with greater tumor specificity, but success was limited, and, again, most researchers abandoned their research in finding a virus to kill cancer in the 1970s and 1980s. [2]

ResurgenceHowever, more recently, there has been a resurgence of interest in finding viruses that can be used to target and attack tumors that have already formed. Some of these modifiied virusesbut not allare known as oncolytic viruses and represent a promising approach to treating cancer. And today, the development of oncolytic viral therapies, which represents a unique therapeutic paradigm within Immuno-Oncology, is rapidly gaining momentum.

In November 2005 research and development of oncolytic viruses got a welcome boost when Chinese medical regulators approved Shanghai Sunway Biotechs genetically modified adenovirus oncorine (H101). This drug was the worlds first oncolytic viral therapy for the treatment of nasopharyngeal carcinoma in combination with chemotherapy after the phase III clinical trial. At the same time, the company also bought the rights to Onyx-15 (dl1520), an almost identical oncolytic virus* developed by Onyx Pharmaceuticals which was scheduled to be included into phase III clinical trials for lung cancer in 2000.

Onyx-15The development of Onyx-15 began in 1996 when Frank McCormick***, Ph.D. FRS, the co-founder of Onyx Pharmaceuticals and one of the companys biochemists, initiated and led various drug discovery efforts.

McCormick believed that an adenovirus without its E1B gene, which inactivates the host cells p53 gene, would be able to selectively kill cancer cells. He believed that because normal cells harboring the modified virus, the defenseless virus would be subject to p53-mediated cell cycle arrest, preventing the virus from replicating. Cancer cells lacking p53 would be thus be unable to halt viral replication and would be lysed, with multiplied viruses bursting out to infect and destroy the entire tumor.

Unfortunately, while McCormicks hypothesis seemed brilliant, it was also incorrect. Research shoeed that Onyx-15 was not specific for p53-null cells. However, in early human trials, the oncolytic virus still killed tumor cells preferentially and was superior to chemotherapy alone. In its phase III trial with oncorine, Shanghai Sunway Biotech reported a 79% response rate for oncorine plus chemotherapy, compared with 40% for chemotherapy alone.

While various key opinion leaders and experts believe that a completed phase III trial of Onyx-15 would have resulted in FDA approval, the development and phase III clinical trial of the oncolytic virus was suspended when Pfizer acquired Warner-Lambert, Onyxs development partner.

Looking back, if approved, Onyx-15 would, as some experts suggest, been made obsolete by better oncolytic viruses. One of the reasons is that the deletion of E3 limited the potency of Onyx-15. But the suspension of the phase III trial stigmatized the development of oncolytic viruses because, as a result, many researchers inaccurately assumed that Onyx-15 trial failed. In reality, the trial never started.

MelanomaIn 2015, the U.S. Food and Drug Administration (FDA) approved the first oncolytic virus immunotherapy for the treatment of cancer called talimogene laherparepvec or T-VEC (Imlygic; Amgen). The treatment is a genetically modified oncolytic viral therapy indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery. Talimogene laherparepvec involves a herpes virus that has been engineered to be less likely to infect healthy cells as well as cause infected cancer cells to produce the immune-stimulating GM-CSF protein.

While in clinical trials and following regulatory approval oncolytic viruses have indeed met the challenge and have become a valuable tool in the anti-cancer armamentarium, there is still much work that needs to be done in the development of novel immunotherapeutic agents based on this approach.

AgreementThe Research Institute has granted an exclusive option to AmunBio covering intellectual property and technology related to an immunotherapeutic oncolytic virus platform. This technology forms the core of AmunBios proprietary, multimodal therapeutic approach utilizing direct destruction of cancer cells and immune system activation.

The technology is based on more than 20 years of research by Prem Seth, Ph.D., Director, Gene Therapy Program at NorthShore, and AmunBios founder and Chief Scientific Officer, and his associates.

AMUN-003The agreement between AmunBio and NorthShore University HealthSystems Research Instituteincludes the development of AMUN-003, an adenovirus-based immunotherapeutic Immuno-Oncology agent, which is being developed for the treatment of multiple solid tumors, including (triple-negative) breast cancer (TNBC) and melanoma, and the ongoing development of additional immunotherapeutic oncolytic viruses.

Following a planned Investigational New Drug (IND) application, which is expected in late 2021/early 2022, AmunBio is planning multiple Phase I clinical trials of AMUN-003 alone or in combination with checkpoint inhibitors.

AmunBios AMUN-003 blocks suppression of the immune response inside the tumor stimulates the recruitment of cancer-killing immune cells and avoids non-specific inflammation. AMUN-003 can be administered both locally as well as systemically.Preclinical studies with AMUN-003 in breast cancer alone, compared to prior oncolytic virus constructs, demonstrated near-complete breast cancer inhibition. [3]

Based on preclinical studies, we believe that AMUN-003 may lead to long-term protection from cancer recurrence, Seth said.

Incidence of cancerWe believe that in the treatment of cancer, there are still not enough new options, noted Cecilia Zapata-Harms, MS, MHA, AmunBio Chief Operations Officer.

While last year, in 2020, the world focused on the SARS-CoV-2 pandemic, more than 9 million people worldwide lost their fight against cancer. *** [4] Among them were more than 600,000 Americans. Our novel viral immunotherapeutic approach may result in significantly more efficacious treatment options, promising to dramatically improve the outcome for many patients diagnosed with cancer, she observed.

We are delighted to be partnering with NorthShore and to be part of ongoing research initiatives to improve the lives of patients, said Zapata-Harms, commenting on research collaboration with the Research Institute.

Breakthrough researchThe Research Institute is the research arm of NorthShore University and supports the organizations core mission to preserve and improve human life through academic excellence and innovative research. The scientists at the research are involved in a wide range of research activities, from lab-based translational research and advanced imaging to clinical trials. As part of this, NorthShore providing the infrastructure and resources for research, which include both administrative and regulatory oversight of investigative studies. The organization has a long history of firsts.

Were humbled by the fact that the same institution, where in the early 1920s George Frederick Dick, MD, and Gladys Henry Dick, MD pioneered the development of a toxin for the prevention of scarlet fever, stands behind our work in developing engineered oncolytic virus for Immuno-Oncology. Its also the same institution where the American pediatrician Louis Wendlin Sauer, MD, in the 1930s, perfected a vaccine used to prevent pertussis (whooping cough), and, more recently, scientists have developed novel treatment options for diseases like MRSA, reducing infection rates through the study of preventative screenings and diagnostic testing, said Andrei R. Shustov, MD, a member of AmunBio Scientific Advisory Board.[5][6]

Oncolytic viruses represent a unique therapeutic approach within Immuno-Oncology and AmunBio platform technology is expected to result in a fundamental shift, augmenting novel treatment modalities compared to, what we believe, is possible today, said, Stephen R. Wachtel, Ph.D., Assistant Vice President, Research Operations at NorthShore.

With the collaboration between the Research Institute and AmunBio were able to address many of the challenges that have prevented previous endeavors from delivering on the full potential of immunotherapeutic oncolytic viruses within Immuno-Oncology, allowing us to discover and develop novel drug candidates for some of the most challenging cancers, Wachtel concluded.

Note*The only difference between the two oncolytic viruses is a slightly larger deletion in H101s E3 gene, which affects immune response**Worldwide, an estimated 19.3 million new cancer cases (18.1 million excluding nonmelanoma skin cancer) and almost 10.0 million cancer deaths (9.9 million excluding nonmelanoma skin cancer) occurred in 2020. Around the world, much work is being done to develop new treatment options.***Today Frank McCormick, Ph.D. FRS, professor in the University of California, San Francisco (UCSF) Helen Diller Family Comprehensive Cancer Center

Clinical trialsSystemic Chemotherapy Combined With Recombinant Human Adenovirus Type 5 and Endostatin Injections for Treatment Malignant Hydrothorax in NSCLC Patients NCT02579564Intraperitoneal Injection of Oncolytic Viruses H101 for Patients With Refractory Malignant Ascites NCT04771676

Highlights of prescribing informationTalimogene laherparepvec (Imlygic; Amgen, Inc) [Prescribing Information]

Reference[1] Kelly E, Russell SJ. History of oncolytic viruses: genesis to genetic engineering. Mol Ther. 2007 Apr;15(4):651-9. doi: 10.1038/sj.mt.6300108. Epub 2007 Feb 13. PMID: 17299401.[2] Data on file AmunBio[3] Sung H, Ferlay J, Siegel RL, Laversanne M, Soerjomataram I, Jemal A, Bray F. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2021 Feb 4. doi: 10.3322/caac.21660. Epub ahead of print. PMID: 33538338.[4] Dick GF, Dick GH. Scarlet Fever. Edinb Med J. 1934 Jan;41(1):1-13. PMID: 29645766; PMCID: PMC5314226.[5] Pittman M. History of the development of pertussis vaccine. Dev Biol Stand. 1991;73:13-29. PMID: 1778306.

Featured image: A microscopic view of a virus with a depth of field. Photo courtesy: 2020 2021 AmunBio, Inc. Used with permission

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AmunBio and NorthShore University to Advance Cancer Immunotherapy with Engineered Oncolytic Viruses - OncoZine