header logo image


Page 43«..1020..42434445..5060..»

Archive for the ‘Gene therapy’ Category

Save the Date: Alliance for Cancer Gene Therapy 2020 Cancer Summit to be Held in New York City – Yahoo Finance

Friday, February 7th, 2020

Thought leaders in cancer cell and gene therapy research will share the latest advances, address the greatest challenges and showcase the most innovative programs in progress today.

Alliance for Cancer Gene Therapy (ACGT) will host its inaugural ACGT 2020 Cancer Summit on April 16, 2020 at the Alexandria Center for Life Science in New York City. The ACGT 2020 Cancer Summit which launches ACGTs 20th anniversary will bring together researchers, companies, investors and advocates in cancer cell and gene therapy to discuss the latest advances, with a focus on combating solid tumors.

A partial list of ACGT 2020 Cancer Summit speakers includes:

Presenting sponsors include Alexandria Real Estate Equities, Inc./Alexandria Venture Investment. Additionally, STAT will be a media partner for the ACGT 2020 Cancer Summit. For more sponsorship information and early bird registration, please visit https://summit2020.acgtfoundation.org

Media Registration

Media registration is free of charge for all valid press card holders or via provision of formal journalist credentials. Register early by contacting ACGT.pr@HDMZ.com to begin receiving advance meeting materials, media alerts, and access to meeting presenters.

About Alliance for Cancer Gene Therapy

For nearly 20 years, Alliance for Cancer Gene Therapy (ACGT) has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. Founded by Barbara and Edward Netter after their daughter-in-laws death from breast cancer, ACGT funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. One hundred percent of all public funds raised by ACGT directly support program and research, thanks to separate funding to support administrative expenses.

For more information, visit acgtfoundation.org, call 203-358-5055, or join the ACGT community on Facebook, Twitter, Instagram and YouTube.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200203005084/en/

Contacts

Media Inquiries: Emily Maxwell 312-506-5220emily.maxwell@hdmz.com

Continued here:
Save the Date: Alliance for Cancer Gene Therapy 2020 Cancer Summit to be Held in New York City - Yahoo Finance

Read More...

Novartis and Gilead provide funding to Vineti – BioPharma-Reporter.com

Friday, February 7th, 2020

The two big pharma companies joined, among others, the investing group for Vineti, the developer of a digital platform for personalized therapeutics enabling the distribution of regenerative medicine, both at clinical and commercial scale.

More specifically, the cloud-based digital platform connects healthcare providers, suppliers, and biopharmaceutical developers by providing them with real-time access to data.

Focusing on personalized medicine, the platform is designed to provide simplicity to end users. Vinetis CEO, Amy DuRoss explained to us why simplicity is important for such treatments:Cell and gene therapies have many, many different types of people involved in the manufacturing and delivery process, including healthcare providers who arent typically part of manufacturing supply chains.

These unfamiliar, highly regulated processes need to be as simple for healthcare providers and other stakeholders as possible, so that these therapies can be produced for patients quickly and without error, she added.

According to the company, Vineti is the only platform of record currently enabling both clinical and commercial personalized therapeutics, such as chimeric antigen receptor (CAR) T-cell therapies and allogeneic cell therapies, into the supply chain.

Both Gilead and Novartis hold assets at commercial stage, the marketing of which can benefit from the use of the platform. Such assets are Novartis gene therapy Zolgensma (onasemnogene abeparvovec-xioi) and Kymriah (tisagenlecleucel), as well as Gileads Yescarta (axicabtagene ciloleucel).

Vinetis Series C financing round closed at $35m (31.8m), with the software developer announcing that it will use the funding for the expansion of its business, including new software product development and commercial expansion in Europe and Asia-Pacific.

The financing was led by Cardinal Health, with participation from Novartis and Gilead, through its subsidiary, Kite, as well as existing investors. Following the completion of the funding, representatives of Novartis and Kite will join Vinetis leadership as board observers.

Kites global head of technical operations, Charles Calderaro, stated that Kite played a foundational role as Vineti's first biopharma partner, when the company prepared for the launch of Yescarta.

Asked about how the platform will be expanded after the funding, DuRoss explained that it will offer improved configurability, enabling users to add new or updated features and adjust them at will, ultimately saving them cost and time.

In biopharma, its all about speed to market, and our configuration approach supports that, DuRoss told us.

Moreover, the platform will position the company towards global expansion, by including more languages as well as support for more regional regulations and standards.

According to DuRoss, the companys partnersincreasingly operate worldwide, in international medical centers and manufacturing sites, and many patient-based drug products cross borders on their journey to becoming a therapy.

Additionally, the company plans to add to the platform more of the standards and integrations required to industrialize cell and gene therapies. Vinetis CEO told us that It may sound contradictory to standardize such deeply personalized drug products, but for scale, standardization is absolutely essential.

DuRoss claimed that even though its still early days in cell and gene therapy, some standard approaches are emerging, as are pre-built integrations that connect critical technology stacks.

We want to help our partners focus on the uniqueness of their science, and not have to reinvent the wheel on supply chain management with every new drug product, she added.

Finally, the company plans to expand the range of therapies that the platform supports to include other high-value therapeutics. Well keep our focus on cell therapy, gene therapy, and personalized cancer vaccines, but expand into other therapeutic areas requiring precision coordination, said DuRoss.

Read more:
Novartis and Gilead provide funding to Vineti - BioPharma-Reporter.com

Read More...

Aperio Hires CAR-T Trials Expert Jonathan Yusi to Support Cell and Gene Therapy Studies – Business Wire

Friday, February 7th, 2020

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Aperio Clinical Outcomes, a leading clinical research organization (CRO), announced today that Jonathan Yusi, an expert in the coordination and management of cell and gene therapy clinical trials, has joined the company as Senior Clinical Trials Manager to support their biotechnology clients in the immuno-oncology space.

Yusi has been managing immune-based therapy trials for over seven years. Prior to joining Aperio, he was a program manager for CAR-T studies and oversaw the first CAR-T program at a large CRO. He has provided independent trial management consulting for CAR-T trials, and his expertise has resulted in lasting KOL relationships within the immuno-oncology space. In addition to his adoptive cell therapy knowledge, Yusi brings over 20 years of clinical research experience to Aperio, with a focus on trial logistics, management, and monitoring of targeted and immune therapies in oncology trials.

Says Suzanne Kincaid, Aperios COO and an oncology industry veteran herself, FDA expects to see over 200 INDs for cell and gene therapies in 2020, so it is imperative that our biotech clients have expertise like Jonathans to manage their trials. He has a strong understanding of the complexities of cell and gene therapy studies and can break down the components for ideal study set-up. We are so excited to have Jonathan help our immuno-oncology clients as they explore these groundbreaking treatments.

Cell and gene therapy trials are a logistical maze, and one missed endpoint can be catastrophic to the study, says Yusi. These programs allow me to utilize everything Ive learned about clinical research and oncology, and my medical and scientific background brings an understanding to the science behind the treatments. The bulk of my career has involved oncology trials, so as the treatments have evolved and become more personalized, my experience has evolved as well. These are life-saving breakthroughs, and Im happy to bring this experience to Aperio and our immune-based therapy clients.

About Aperio Clinical Outcomes

In a data driven industry, Aperio remains dedicated to transparency with clients and focused on the most important part of the process: people. Aperio provides full, customizable clinical research services across multiple therapeutic areas, as well as consulting services in Quality Assurance, Strategic Resourcing and Clinical Trial Technology. For more information: http://www.aperioclinical.com.

View original post here:
Aperio Hires CAR-T Trials Expert Jonathan Yusi to Support Cell and Gene Therapy Studies - Business Wire

Read More...

Advances in Cell and Gene Therapy and Opportunities in China – BSA bureau

Friday, January 17th, 2020

GenScript Biotech Global Forum Highlights Advances in Cell and Gene Therapy and Opportunities in China

GenScript Biotech Corp., a leading global biotechnology group and a pioneer in the field of gene synthesis, held its inaugural "Global Forum on Cell & Gene Therapy and the Booming China Market," during the JP Morgan Healthcare Conference week, attracting hundreds of industry leaders, investors and others to address the challenges and opportunities in this innovative field.

"As an industry, we are on the brink of achieving some extraordinary breakthroughs in cell and gene therapy for cancer and other diseases," said GenScript Biotech CEOFrank Zhang, PhD. "Four gene and cell therapies have recently been approved by the FDA, bringing new hope to patients, and this is only the beginning. Our vision is to make cancer a chronic or curable disease rather than a deadly one, and to transform the treatment of cancer, autoimmune and other diseases by leveraging the advantages of cell and gene therapy."

While significant advances are being made, the Forum also tackled some of the more pressing challenges, such as mitigating treatment side effects, improving treatment efficacy in solid tumors and scaling up manufacturing. Panelists from Kite Pharma, GE Healthcare Life Sciences, Ziopharm Oncology, Oxford Biomedica, Genethon, CARsgen Therapeutics, J&J Innovation Asia Pacific, the American Society of Gene & Cell Therapy, Loncar Investment, Lilly Asia Ventures, and many others participated in the event.

In the U.S. alone, the U.S. Food and Drug Administration is expected to approve 40-60 cell and gene therapies by 2030. During a panel discussion focused on regulatory issues, experts considered what regulators will need to do to keep up with the rapid pace of innovation, the new hospital-based regulatory pathway inChina, how to ensure quality through the manufacturing process, and the challenges and opportunities that come with regulatory harmonization among different countries.

Chinacontinues to attract significant attention from industry and investors and is poised to grow even more. During his welcoming remarks, Zhang notedChina'semergence as a global economic leader, with a projected$1.1 trillionspend on healthcare this year, as well as the growing disease burden inChina. By 2030, an estimated 4.3 million Chinese will be diagnosed with one of the 14 major cancers, according to research from IMS Health. Panelists addressed issues such as the amount of capital required to achieve scale inChina, and advantages of the market inChina.

"The drug development business is changing rapidly andChinais at the fore in a number of ways," Zhang said. "Biotech and pharma companies do not need or desire to have the infrastructure to scale their drugs through commercialization. With lower costs,Chinais a natural place for companies to contract out costly development and manufacturing to organizations that have the expertise and experience to collaborate with them through the entire discovery to development lifecycle."

For its part, GenScript has put significant resources into its Contract Development and Manufacturing Organization (CDMO) business to meet the increasing demand. In 2018, the company officially launched its biologics CDMO segment, and last year opened a new GMP compliant biologics research center. GenScript is also leading the way in cell therapy through its antibody discovery service and plasmid and virus production capabilities.

Read more:
Advances in Cell and Gene Therapy and Opportunities in China - BSA bureau

Read More...

UK accounts for 12% of cell and gene therapy trials – report – – pharmaphorum

Friday, January 17th, 2020

The UK accounts for over 12% of global cell and gene therapy clinical trials, according to new data published by a government-backed agency.

The Cell and Gene Therapy Catapult (CGT Catapult) today released its 2019 UK Advanced Therapy Medicinal Products (ATMPs) clinical trials database showing that the 127 ongoing trials represent a 45% increase compared with 2018.

According to the CGT Catapult this means the NHS and UK life sciences R&D centres are working well to allow innovative therapies to progress through to the clinic.

Publication of the figures comes the day after the government announced a deal with Novartis to bring cholesterol drug inclisiran to patients in England, and the announcement of an updated life sciences industrial strategy aimed at stimulating private investment in the sector.

Cell and gene therapies require specialist systems and infrastructure and according to the CGT Catapult the UK is being recognised internationally with the majority of commercially sponsored trials being backed by international organisations.

The database shows that 77% of UK cell and gene therapy trials are now sponsored by commercial organisations compared to only 25% in 2013.

The main therapy area for cell and gene therapies clinical trials remains oncology (39%) followed by ophthalmology (13%) and haematology (12%).

While the majority of trials are in the recruitment phase, the number of trials recruiting is considerably larger than previous years.

In 2019, there were 90 cell therapy trials recruiting patients, compared with around 55 in 2018, and the data suggest that these trials are quickly moving through planning and regulatory approvals to recruitment stage.

Around 65% of trials involve autologous cells, sourced from individual patients, with the remainder being off the shelf allogeneic products.

Established as an independent centre of excellence the CGT Catapult is funded by the government through Innovate UK.

It aims to encourage inward investment from big pharma and other international companies by bridging the gap between scientific research and full-scale marketing.

Keith Thompson, CEO of the Cell and CGT said: The total number of cell and gene therapy clinical trials in the UK has been increasing consistently by an average of 25% year on year since 2013. This has been enabled by the development of the UKs fantastic ecosystem to support the development and clinical adoption of cell and gene therapies.

The full report is available here.

Continue reading here:
UK accounts for 12% of cell and gene therapy trials - report - - pharmaphorum

Read More...

Why gene therapy is set to transform medicine – Digital Journal

Friday, January 17th, 2020

Gene therapies are in the in-demand medicine, due to their potential to treat life-threatening diseases, including illnesses classified as rare genetic diseases. Examples include treating sickle cell anemia and anti-tumor immunotherapy. As well as being demand, the market is also lucrative, expected to grow to $11.96 billion by 2025. The growth with gene therapies is represented by the range of gene therapy products in the clinical pipeline, where Frost and Sullivan estimate some 400 cell and gene therapy products in development. The last year alone has seen a 27 percent increase in the number of clinical trials involving gene therapy products. This is also reflected with the number of emergent start-up companies entering the gene therapy space. Most start-up companies elect to outsource the downstream manufacturing stages, due to the relatively low costs involved, as off-set against the costs involved with constructing specialist facilities. Big Pharma is also investing in the field. For example, Pfizer has injected $500 million into one of its plants in North Carolina and Fujifilm Diosynth has made a similarly large investment to boost production capability. READ MORE: Revolutionizing the CRISPR methodThe growth has been fuelled by support from governments and regulatory agency. Here the U.S. 21st Century Cures Act as been a key driver, supported by the U.S. Food and Drug Administration (FDA). There are, however, some limitations to be overcome. One limitation with progressing gene therapy products is with costs and pricing structures, which is resulting in many therapies being unstainable. This is also limiting the transition to scale, given the complexities tied to growing, sourcing and transporting cells. Costs are high partly as a result of the development and partly because many gene therapies offer curative solutions, making them an in-demand and expensive medicine. A solution to the costs factors is, according to Vered Caplan (Orgensis Inc) the development of point-of-care automation, including the application of closed systems for processing cell therapies. These systems require the use of fewer personnel and permit real-time quality control testing to take place. A second limitation is with the availability of rare materials of sufficient quality, such as plasmid DNA and tranfection agents), which need to be manufactured under the same standards applied to mainstream pharmaceuticals (that is, Good Manufacturing Practices. With new technology emerging to improve product yield and to drive down costs, coupled with increased investments, the expansion of gene therapy products looks set to be one of the big drivers within healthcare over the next five years.

Follow this link:
Why gene therapy is set to transform medicine - Digital Journal

Read More...

Precision BioSciences hits two key FDA milestones in advancing gene therapy for cancer – WRAL Tech Wire

Friday, January 17th, 2020

Precision BioSciences, a Durham-based genome editing company, has reached two regulatory milestones for its potential therapy against multiple myeloma, a chronic cancer of white blood cells.

The U.S Food and Drug Administration (FDA) has accepted the companys Investigational New Drug (IND) application and also granted the therapy Orphan Drug Designation, a status that gives companies tax reductions and other incentives to develop treatments for rare diseases.

The therapy, designated as PBCAR269A, is Precisions third allogeneic chimeric antigen receptor (CAR) T cell therapy candidate.

FDA acceptance of the IND for PBCAR269A further underscores the ongoing progress in our allogeneic CAR T pipeline, said Matt Kane, co-founder and chief executive officer of Precision BioSciences. We have now moved three CAR T programs from preclinical to clinical stage development since April 2019, and we look forward to continuing to advance our allogeneic CAR T portfolio to bring these novel therapeutic candidates to patients.

Matthew Kane

The company plans to begin a Phase 1 clinical trial of the therapy this spring at multiple sites using material produced at its own manufacturing facility in Durham. About 48 patients are expected to be enrolled.

For more information about the trial, visitwww.clinicaltrials.gov, and enter study identifier number NCT04171843.

In preclinical disease models, PBCAR269A has demonstrated no evidence of graft-versus-host disease at doses that resulted in potent anti-tumor activity, said Chris Heery, M.D., chief medical officer of Precision BioSciences. There remains significant unmet need in the treatment of relapsed/refractory multiple myeloma, and we are excited to begin clinical trials with an off-the-shelf CAR T therapy candidate in this setting.

The IND for PBCAR269A builds on the initial clinical data Precision presented in late 2019 for its lead program, a CAR T therapy for treating non Hodgkins lymphoma and B-cellacute lymphoblastic leukemia, and the FDAs acceptance of an IND for another CAR T therapy for treating non-Hodgkins lymphoma, chronic lymphocytic leukemia and small lymphocytic lymphoma.

Precision BioSciences harnesses T cells, a type of white blood cell that is vital to the adaptive immune systems ability to identify specific antigens and destroy pathogens. Through the companys CAR T technology, the T cells can be directed to kill cancer cells.

Precision produces the CAR T therapies by selecting T cells derived from healthy donors as starting material. Then, using its proprietary ARCUS genome-editing technology, the company modifies the donor T cells.

Scientists insert the CAR gene at the T cell receptor locus, enabling the T cell to target a specific marker on a cancer cell, while knocking out the T cell receptor to prevent the patients immune system from recognizing and attacking the T cells.

The company optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of certain types of CAR T cells throughout the manufacturing process and in the final product.

The process creates a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease, normally a major challenge when inserting foreign or altered cells or tissues into the body.

The company has posted a four-minutevideoon its website to explain CAR T therapy, using Samurai warriors as an analogy.

Last July Precision opened its Manufacturing Center for Advanced Therapeutics (MCAT), the first in-house current Good Manufacturing Process (cGMP)-compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor CAR T cell therapy products.

Precision Bio facility

Given the potential output of our platform, weve known from the beginning that it was critical for us to address the need for scalable manufacturing of cell-therapy products in order to be able to effectively deliver them to patients, Kane said when the facility opened. In addition to our clinical work, it also has the potential to be a commercial launch facility with the capacity to generate up to 10,000 doses of CAR T cell therapies and 4,000 doses of gene therapies per year.

The facility can produce three different drug substances: allogeneic CAR T cells, messenger RNA and adeno-associated viral vectors. It was designed to meet regulatory requirements in the United States, Europe and Japan.

In addition to health care, Precisions ARCUS genome-editing platform has applications in food and agriculture.

In 2018 the company created a new name and brand identity, Elo Life Systems, for its food and agriculture business, previously known as Precision PlantSciences, based in Research Triangle Park.

Elo is using the ARCUS platform and other new technologies for applications in crop improvement, animal genetics, industrial biotechnology and sustainable agriculture.

Since it was spun out of Duke University in 2006, Precision raised about $300 million in venture capital, government grants and collaboration agreements. The company went public in March 2019, grossing $145.4 million in an initial public offering of stock.

The companys shares are listed on the Nasdaq Global Select Market under the ticker symbol DTIL, shorthand for the companys marketing tagline, Dedicated to improving life.

(C) N.C. Biotech Center

Original post:
Precision BioSciences hits two key FDA milestones in advancing gene therapy for cancer - WRAL Tech Wire

Read More...

ASC Therapeutics and Vigene Announce Long-Term Strategic Partnership for Gene Therapy Development and Manufacturing – Yahoo Finance

Friday, January 17th, 2020

ASC Therapeutics ("ASC"), a privately-held gene therapy company focused on developing transformative gene-based medicines for serious diseases, announced today that it has entered into a long-term strategic manufacturing partnership with Vigene Biosciences ("Vigene"), a Maryland-based Contract Develop and Manufacturing Organization (CDMO). Vigene will provide ASC with access to GMP manufacturing including viral vectors and plasmid DNA for its hemophilia A gene therapy clinical program, as well as a manufacturing platform for future gene therapy programs.

"The genetic platform technology developed by ASC is going to change the way serious diseases are treated in the future. We have seen remarkable potency data in our Hemophilia A IND-enabling gene therapy studies," said Dr. Ruhong Jiang, ASC Founder & CEO.

Dr. Jiang continued, "We are proud to welcome Vigene, a global leader in gene therapy process development and GMP manufacturing with a proven track record and expertise in the field of viral vector manufacturing, to become an integral part of ASC long-term plan. Our partnership provides ASC access to Vigenes world-class team with expertise for both plasmid DNA and viral vector manufacturing as well as high-caliber QC and QA teams."

"Vigene is excited to become a long-term CMC partner for ASC with multiple newly built, state-of-the-art GMP suites and high-titer virus producer cell lines, we are well positioned to support ASC for both early-stage and commercial virus production. This partnership will ensure that all ASC clinical deliverables are achieved in a timely fashion," said Dr. Zairen Sun, Vigenes President and CEO. "We have a world-class manufacturing team, and this agreement is a testimony for our recognition by top-tier biopharmaceutical companies."

About ASC Therapeutics

ASC Therapeutics is a biopharmaceutical company dedicated to advancing an integrated and sustainable pipeline focused on gene therapies that correct the root cause of complex and intractable diseases. We are leveraging our AAV-based gene therapy, CRISPR-Cas9 and proprietary gene editing platforms consolidated over 10 years to develop transformative gene-based medicines. ASC is accelerating through clinical trials several lead gene therapies for inherited blood disorders. Our team of industry veterans in discovery, pre-clinical, clinical and CMC gene therapy development and world-class academic and biopharmaceutical partnerships are potentiating our gene therapy capabilities. To learn more about ASC Therapeutics, please visit http://www.asctherapeutics.com.

About Vigene Biosciences

Vigene Biosciences is an award-winning and private equity backed leader in gene delivery development and manufacturing. Vigene has 16 years of cGMP viral vector production experience. Vigenes mission is to make gene therapy affordable. Vigene offers IND-enabling and IND-supporting materials as well as FDA- and EMA-compliant commercial products for plasmid, AAV, lentivirus, and adenovirus with proven technologies and track records. The GMP facility features 10 GMP suites including 5 brand-new cGMP suites with 200L-500L single-use stir tank bioreactors for suspension cells as well as iCellis 500 for adherent cells. In 2018 and 2019 Vigene was recognized by INC500. In 2018, Vigene was chosen as the ACG Emerging Company of the Year Award. To learn more about Vigene Biosciences, please visit http://www.vigenebio.com

View source version on businesswire.com: https://www.businesswire.com/news/home/20200114005788/en/

Contacts

ASC Contact Information: Oscar Segurado, MD, PhDChief Medical OfficerASC TherapeuticsMilpitas, CA650.490.5199oscar.segurado@asctherapeutics.com

Vigene Contact Information: Jeffrey Hung, Ph.D.Chief Commercial OfficerVigene BiosciencesRockville, MD301.251.6638jhung@vigenebio.com

Link:
ASC Therapeutics and Vigene Announce Long-Term Strategic Partnership for Gene Therapy Development and Manufacturing - Yahoo Finance

Read More...

One-year Results in 2 Given Gene Therapy at Low Dose Showing… – Parkinson’s News Today

Friday, January 17th, 2020

Two Parkinsonspatients treated with AXO-Lenti-PD, an investigative gene therapy, in an ongoing clinical trial continue to show improvement 12 months later, Axovant, the therapys developer, said in a release.

These findings at one year after treatment are important because this timepoint allows for a better assessment of therapy durability, and a more assured differentiation between placebo effects and therapeutic response, the company added.

AXO-Lenti-PD has shown encouraging results in these two people given a first low dose in the SUNRISE-PD (NCT03720418) Phase 1/2 clinical trial, which is now enrolling up to 30 patients at sites in France and England.

The treatment works by delivering three genes involved in dopamine production directly to the brain via a surgical procedure.

Dopamine is a neurotransmitter a molecule involved in transmitting information between neurons that is critical to coordinating movement. Dopamine-producing (dopaminergic) neurons are lost in Parkinsons, and the resulting drop in dopamine levels is the cause of many disease symptoms.

By infecting brain cells with the genetic instructions to increase dopamine production, AXO-Lenti-PD aims to turn other cells into dopaminergic neurons.

Current dopamine replacement therapies require continual oral doses of dopamine, whose effectiveness fades over time. The period between when one doses effectiveness wanes and the taking of a next dose can result in off periods, wherein patients report a return of symptoms such as poor motor control, stiffness, fatigue and mood changes.

Helping the brain to again produce adequate levels of dopamine would, in theory, eliminate the need for periodic oral doses, which could significantly limit off periods.

Previous studies in primate models of Parkinsons found AXO-Lenti-PD to be safe and effective, and SUNRISE-PD results at three months post-treatment found that a one-time delivery of the therapy significantly improved patient scores on theUnified Parkinsons Disease Rating Scale (UPDRS), a standard assessment of motor and non-motor symptoms associated with Parkinsons.

The trial consists of two parts. Part A is an open-label, dose-escalation phase in which patients receive one of potentially three escalating doses of the gene therapy. In part B, a new group of patients will be randomized to either the ideal part A dose or to a sham procedure as an untreated control group. SUNRISE-PDs goal is to test the safety, tolerability, and effectiveness of the potentialtreatment.

Both patients here, the first two enrolled, received the lowest dose (4.2106transducing units) of AXO-Lenti-PD.

One-year results showpositive changes of 24 points and 20 points (respectively for the two patients) on the UPDRS Part III Off score, representing a 37% improvement in off-period motor symptoms, Axovant reported. Improvement at six months was 29%, as measured on the same scale.

These patients also showed an average 13-point positive change from baseline (study start) representing a 44% improvement on the UPDRS Part II Off score, which assesses daily life activities. On the PDQ-39 score index, another quality-of-life measure in Parkinsons disease, these two showed an average 15-point positive change, or a 30% improvement from baseline to 12 months.

Both patients tolerated AXO-Lenti-PD well, and neither reported any serious side effects. One maintained a diary of on/off periods, which is useful in evaluating changes that might be due to therapy across time.

People being enrolled in SUNRISE-PD have had Parkinsons for at least five years, have motor fluctuations and dyskinesia (jerky, involuntary movements), and are between the ages of 48 and 70. More information can be found here.

The company expects to soon release six-month results on the first two patients given a second and higher dose of AXO-Lenti-PD. This dose is three times higher than that given the first cohort.

If dose-escalation results allow, Axovant expects to begin the randomized and placebo-controlled part B of the SUNRISE-PD as a Phase 2 study by the close of 2020.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.

Total Posts: 208

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

Here is the original post:
One-year Results in 2 Given Gene Therapy at Low Dose Showing... - Parkinson's News Today

Read More...

Global Gene Therapy Market 2019-2030: Promising Therapeutics Areas – Yahoo Finance

Friday, January 17th, 2020

Dublin, Jan. 13, 2020 (GLOBE NEWSWIRE) -- The "Gene Therapy Market (3rd Edition), 2019-2030" report has been added to ResearchAndMarkets.com's offering.

The Gene Therapy Market, 2019-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, and genome editing therapies. The study also features an elaborate discussion on the future potential of this evolving market. Amongst other elements, the report features:

Key Topics Covered:

Some of the Companies Mentioned include:

For more information about this report visit https://www.researchandmarkets.com/r/nuaak0

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

CONTACT: ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.comFor E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

Read more:
Global Gene Therapy Market 2019-2030: Promising Therapeutics Areas - Yahoo Finance

Read More...

What has the most impact on longevity? – Harvard Health

Friday, January 17th, 2020

Published: February, 2020

Q. My family tends to be long-lived. I hear longevity is due to our DNA, and I also hear it's due to lifestyle. Which is it, and how do they make us live longer?

A. Both DNA and lifestyle can affect longevity, and they both do so in the same way: by altering our body chemistry. DNA controls the production of each of the natural chemicals in our body. It controls both the shape (and, hence, the effectiveness) of each chemical, and also controls how much of that chemical is made. So, it's not surprising that DNA could affect longevity. In the past 20 years, astonishing progress has been made in understanding the body chemistry that controls the aging process. And that knowledge has allowed scientists to extend the life of various animals through simple genetic manipulations.

Subscribe to Harvard Health Online for immediate access to health news and information from Harvard Medical School.

Originally posted here:
What has the most impact on longevity? - Harvard Health

Read More...

Frazier closes 12th fund in 30 years, with $617M to bet on cell/gene therapy, Big Pharma spinoffs and more – Endpoints News

Friday, January 17th, 2020

The team at Frazier Healthcare did a number of deals that exemplified its wide-ranging strategy in 2019: Tachi Yamada worked with gene therapy pioneer Jim Wilson to launch Passage Bio; Mike Gallatin sold Mavupharma and its STING-targeted small molecule to AbbVie; and Bhaskar Chaudhuri flipped Arcutis to an IPO just months after introducing it to the world via a crossover round close to $100 million. Theyre now kicking off 2020 with a new, bigger fund that will give them firepower to do more.

At $617 million, Fund X is fairly dramatically bigger than their last fund, said managing partner Patrick Heron.

Were increasingly going after new therapeutic modalities like gene therapy, cell therapy, neoantigens and because those need significant manufacturing and CMC investment or investing more dollars per company, he told Endpoints News.

He sees Frazier pouring around $40 million into each company supporting them through every stage, whether its helped with the launch or joined through a later syndicate though that could vary if, say, they sell a company right after Series A. By that estimate, the new fund could touch anywhere from 15 to 25 biotechs.

About a third of the portfolio is reserved for homegrown startups, another 15% to 25% for public securities, and the rest is in-between.

Heron is one of three leading the fund; hes joined by managing partner James Topper and Dan Estes, whos just been promoted to general partner. Also involved will be Jamie Brush, newly made partner after spearheading investments in public securities for the past three years.

Frazier is happy to both create and syndicate with its venture brethren, Heron added, citing OrbiMed as a friend.

The congregation has grown exponentially since Frazier first put its feet down three decades ago.

When I started at Frazier, there were probably 10 to 15 life sciences focused VC funds, said Heron, whos just celebrated his 20th year at the firm, and now theres probably 100.

It gives Frazier a lengthy track record to boast which can be particularly helpful when they pitch bigger players on biotech spinoffs such as Phathom Pharmaceuticals, now developing one of Takedas GI drugs.

Pharma companies have become more receptive to that when they see substantial value accrual to them, Heron said. And its public now: Takeda owns probably about $200 million worth of stock in Phathom, and so they are basically deriving a lot of economic value from the partnership, and what theyre also focused on is the quality of teams we can put around their asset such that the program will reach the clinic and benefit patients.

Two former Celgene execs from the global inflammation and immunology franchise have been recruited to the C-suite at Phathom, including CEO Terrie Curran and CCO Martin Gilligan.

Despite the lack of big check M&A at the beginning of the year and an election looming in November, Heron remains optimistic as their deal flow has been in line with the expectation of 2 to 3 sales per year. And the same goes for IPO.

I think you will see a lot of companies sort of backing up the truck and loading up with as much capital as they can, with probably less robust activity in the second half of the year, he said.

Continue reading here:
Frazier closes 12th fund in 30 years, with $617M to bet on cell/gene therapy, Big Pharma spinoffs and more - Endpoints News

Read More...

Ultragenyx shares jump on ‘better than expected’ gene therapy data – FierceBiotech

Sunday, January 12th, 2020

Ultragenyx Pharmaceutical saw its shares jump around 27% in trading Friday after announcing positive top-line data out of its gene therapy trial.

Its a small number, just three patients that form part of a third cohort for the phase 1/2 study, as well as another small test but a longer-term look from the second cohort.

In cohort three testing the biotechs drug DTX301, an adeno-associated virus gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency, there were two confirmed female responders as well a third potential male responder who requires longer-term follow-up to confirm response status.

The Art of Recognizing Clinical Supply Risk Factors and Applying Proactive Measures to Avoid Study Delays and Disruptions

No two studies are the same and each clinical supply project carries unique risks. But what characteristics are most likely to raise a flag that issues are ahead? Are there certain types of clinical sponsors and studies that are at greater risk of experiencing supply challenges? And how do clinical sponsors know what is important to focus on and what is not? Join us for this webinar as we attempt to answer these questions.

Meanwhile, in cohort two, one female patient saw a new response after a year. The biotech added that the two previously disclosed responders in cohort one and two also remain clinically and metabolically stable at 104 and 78 weeks, respectively. Across all nine patients dosed in the study, up to six patients have demonstrated a response, it said in a statement.

RELATED: BIO: In conversation with Emil Kakkis, Ultragenyx CEO

OTC deficiency is a rare X-linked genetic disorder characterized by complete or partial lack of the enzyme OTC. Excess ammonia, which is a neurotoxin, travels to the central nervous system through the blood,

According to the National Organization for Rare Disorders, the severity and age of onset of OTC deficiency vary from person to person, even within the same family. A severe form of the disorder affects some infants, typically males, shortly after birth (neonatal period). A milder form of the disorder affects some children later in infancy. Both males and females may develop symptoms of OTC deficiency during childhood. Most carrier females are healthy, but may be prone to severe headaches following protein intake.

Analysts at Jefferies said the data looked better than expected and could be a positive spark to help turn the stock heading into 2020 events. It certainly did in the immediate term, with the biotechs shares up by 27% in mid-morning trading Friday.

We are encouraged to see a more uniform response at the higher doses including three female responders. To date, three patients in the study have discontinued alternate pathway medication and liberalized their diets while remaining clinically and metabolically stable, said Eric Crombez, M.D., chief medical officer of the Ultragenyx Gene Therapy development unit.

We are moving to prophylactic steroid use in the next cohort as we believe this could further enhance the level and consistency of expression that we have demonstrated so far.

Read this article:
Ultragenyx shares jump on 'better than expected' gene therapy data - FierceBiotech

Read More...

Gene therapy company begins operations in Longmont – The Denver Channel

Sunday, January 12th, 2020

LONGMONT, Colo. A few months ago, 2-year-old Maisie Forest was finally able to sit up on her own for the first time. Her development has been delayed by a rare genetic disorder called Spinal Muscular Atrophy, but last August, she received a groundbreaking treatment for the condition.

"It's a miracle drug," said Maisie's mother, Ciji Green. "It's not the cure, but we're talking about a disease that had no treatments four years ago," she added.

The "miracle drug" Green is referring to Zolgensma, a gene therapy for Spinal Muscular Atrophy made by Novartis-owned AveXis. On Tuesday, AveXis cut the ribbon on a new facility in Longmont where it will soon produce Zolgensma.

"Zolgensma is this first product weve had approved by the FDA for the treatment of kids with Spinal Muscular Atrophy," said AveXis President David Lennon.

The FDA approval came last May, just in time for Maisie to receive the treatment. But her mother still had to fight for the insurance company to pay for it. At $2.1 million per dose, Zolgensma is the most expensive drug or treatment ever made. Lennon said Novartis has invested half a billion dollars in the production of Zolgensma.

For Green, the cost is well worth the changes she's already seen in her daughter. Speaking to employees at the AveXis ribbon cutting, she called them heroes.

"To all of you it may just be a treatment, but to my family and so many others, its so much more," said Green.

AveXis says the same platform they used to produce Zolgensma might be applied to other therapies for other diseases in the future. The company is looking at developing treatments for Rett Syndrome, Friedreichs Ataxia, and an inherited form of Amyotrophic Lateral Sclerosis, or ALS.

"There are actually thousands of these kinds of diseases. Usually they impact a few hundred kids or adults every year, but altogether there are potentially millions of patients who have genetic diseases around the world," said Lennon.

Lennon said AveXis chose Longmont for its production facility in part because of the infrastructure already in place. The building at 4000 Nelson Rd. was previously occupied by pharmaceutical companies AstraZeneca and Amgen. He said the available talent was also a factor.

AveXis retained most of the employees from the previous tenants. With new hires, the Longmont facility currently has a staff of around 300 employees and expects to grow to 400 by the end of 2020.

More:
Gene therapy company begins operations in Longmont - The Denver Channel

Read More...

Scientists want human trials for gene therapy that could help battle addiction – Digital Trends

Sunday, January 12th, 2020

In recent years, new gene editing tools have been used for everything from genetic modification of plants to increase crop yields to, far more controversially, genetic tampering with human embryos. Could a form of gene therapy also be useful in helping treat cocaine addiction, a form of addiction that proves highly resistant to alternative approaches, such as conventional medical treatment and psychotherapy? Thats what researchers from the world-famous Mayo Clinic are hoping to prove.

They are seeking approval for the first-in-human studies of an innovative new single-dose gene therapy. Their approach involves the delivery of a gene coding for an enzyme, called AAV8-hCocH, which metabolizes cocaine in the body into harmless byproducts. In order to progress to this next step in their work, they first have to gain permission from the U.S. Food and Drug Administration (FDA) in the form of an Investigational New Drug Application.

The researchers have already demonstrated the safety of their approach in mice. In a prior experiment, they showed a complete lack of adverse effects in mice which had both been previously exposed to cocaine and those which had not.

Mice given one injection of AAV8-hCocH and regular daily injections of cocaine had far less tissue pathology than cocaine-injected mice with no vector treatment, the researchers wrote in the abstract for their paper describing the work. Biodistribution analysis showed the vector located almost exclusively in the liver. These results indicate that a liver-directed AAV8-hCocH gene transfer at reasonable dosage is safe, well-tolerated, and effective. Thus, gene transfer therapy emerges as a radically new approach to treat compulsive cocaine abuse.

This is not the first time similar work has been carried out. In February 2017, scientists at the University of British Columbia genetically engineered a mouse so as to be incapable of becoming addicted to cocaine. However, one of the researchers on the project told Digital Trends that transferring this work across to humans for possible treatment for addiction was not straightforward. Instead, that work was more focused on exploring the link between drug use and genetics and biochemistry.

Theres still a whole lot more research that needs to be done in this area. Even if the FDA grants the Mayo Clinic researchers permission for their human trials, well most likely be waiting a few years at least before this treatment could be rolled out to the general public. Its an exciting leap forward, nonetheless.

Read the rest here:
Scientists want human trials for gene therapy that could help battle addiction - Digital Trends

Read More...

At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease – The New York Times

Sunday, January 12th, 2020

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared. But doctors had given her blood transfusions while she regrew her own red blood cells, so it was not clear if the absence of symptoms was because of the gene therapy or the transfusions.

As she recovered, Helen returned to her passion: dancing. One day, she came back from her school dance group and told her mother, My legs hurt. It feels funny. Ms. Cintron smiled. Thats soreness, she explained. Helen laughed. She had only known pain from sickle cell.

Helen was scheduled for her six-month checkup on Dec. 16. By then, all the transfused cells were gone, leaving only blood made by stem cells in her own marrow. The doctors would finally tell her whether the therapy was working.

The day before, she and her parents visited the New England Aquarium in Boston. She was able to stay outside on a cold, blustery day, watching one seal bully the others, barking and fighting. When Helen mentioned that her hands were cold, Ms. Cintrons stomach clenched in fear. But it was just a normal thing to feel on a winter day.

The next morning, Dr. Esrick delivered the news. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved even with blood transfusions. She had no signs of sickle cell disease.

Now you are like me, her father told her. I jump in the pool, I run. Now you can do it, too!

Her family, accustomed to constant vigilance, is only now getting used to normal life.

On Dec. 23, Helen and her mother flew to the familys new home in Arizona.

Helen recently described her transformed outlook on Facebook.

View original post here:
At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease - The New York Times

Read More...

Generation Bio grabs a $110M round to ramp up work on next-gen gene therapies – FierceBiotech

Sunday, January 12th, 2020

In 2018, Generation Bio broke cover with a $25 million series A, swiftly followed by a meatier $100 million second funding round.

Now, just before the J.P. Morgan Healthcare Conference, it has grabbed its biggest yet, a $110 million series C, as it looks to go all in for IND-enabling studies for its leading programs: liver-targeted therapies for hemophilia A and phenylketonuria.

In addition to the liver, Generation Bio is also working on potential treatments for diseases of skeletal muscle and the eye.

The Art of Recognizing Clinical Supply Risk Factors and Applying Proactive Measures to Avoid Study Delays and Disruptions

No two studies are the same and each clinical supply project carries unique risks. But what characteristics are most likely to raise a flag that issues are ahead? Are there certain types of clinical sponsors and studies that are at greater risk of experiencing supply challenges? And how do clinical sponsors know what is important to focus on and what is not? Join us for this webinar as we attempt to answer these questions.

The early-stage Cambridge, Massachusetts-based biotech saw its major round led by T. Rowe Price with help from Farallon, Wellington Management and existing investors Atlas Venture, Fidelity, Invus, Casdin, Deerfield, Foresite Capital and an entity associated with SVB Leerink.

Generation Bios platform is geared up to be gene therapy 2.0 and is designed to develop re-dosable, long-lasting, scalable gene therapies for severe diseases.

The company is developing gene therapies under the GeneWave banner that use closed-ended DNA rather than viruses to deliver therapeutic proteins, which could sidestep safety issues such as immune reactions

Our vision is to develop re-dosable, long-lasting gene therapies manufactured at a scale that leaves no patient or family behind, said Geoff McDonough, M.D., president and CEO of Generation Bio.

Since our founding we have had the support of high-quality investors who share our excitement about the potential of our platform to lead a new generation of gene therapy and about advancing our lead programs toward the clinic.

Read the original:
Generation Bio grabs a $110M round to ramp up work on next-gen gene therapies - FierceBiotech

Read More...

Solid Bio Restructures to Get Halted Gene Therapy Study Back on Track – Xconomy

Sunday, January 12th, 2020

XconomyBoston

Solid Biosciences is slashing its workforce, including two top executives, in order to devote the companys remaining resources to its experimental gene therapy for Duchenne muscular dystrophy.

The corporate restructuring announced Thursday comes two months after the FDA placed a hold on the study after safety problems emerged that were linked to the gene therapy, SGT-001. Cambridge, MA-based Solid Bio (NASDAQ: SLDB) says going forward it will focus on how to address the clinical hold and resume testing. With the corporate changes, Solid Bio says it has enough cash to last into next year. At the end of the third quarter of 2019, the company reported cash and other holdings totaling $105.7 million.

Following the announcement, Solid Bios stock price slid more than 17 percent to $3.66 per share in pre-market trading.

Solid Bio has been developing SGT-001 as a way to potentially address the genetic defect underpinning Duchenne. Patients who have the inherited disease dont make enough of the muscle protein dystrophin. The Solid Bio gene therapy uses an engineered virus to deliver genetic material intended to restore dystrophin production. But the company had also previously disclosed theres a chance that the dosing requirements of the gene therapy could increase the risk of side effects related to the virus used in the treatment.

The complications reported in the November clinical hold included an immune system reaction, a decrease in red blood cells, kidney injury, and blood circulation difficulties. Those problems are similar to ones cited in the FDAs 2018 clinical hold on tests of SGT-001. Months later, the agency allowed the study to resume but with additional safety measures.

Solid Bios board approved the corporate restructuring on Tuesday, according to a securities filing. In the first quarter of this year, the company expects to record a $2.1 million charge related to the layoffs, which will cut about one third of its workforce. Last years annual report states that the company had 111 full-time employees as of Dec. 31, 2018. Those leaving Solid Bio include Alvaro Amorrortu, the companys chief operating officer, and Jorge Quiroz, its chief medical officer. But both will continue to advise Solid Bio under consulting agreements.

Photo by Flickr user reynermedia via a Creative Commons license

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan [[at]] xconomy.com.

See the original post:
Solid Bio Restructures to Get Halted Gene Therapy Study Back on Track - Xconomy

Read More...

At 16, shes a pioneer in the fight to cure sickle cell disease at Boston Childrens – Boston.com

Sunday, January 12th, 2020

BOSTON Helen Obando, a shy slip of a girl, lay curled in a hospital bed in June waiting for a bag of stem cells from her bone marrow, modified by gene therapy, to start dripping into her chest.

The hope was that the treatment would cure her of sickle cell disease, an inherited blood disorder that can cause excruciating pain, organ damage and early death.

Helen, who at 16 was the youngest person ever to undergo the therapy, was sound asleep for the big moment.

It was a critical moment in medical science.

For more than a half-century, scientists have known the cause of sickle cell disease: A single mutation in a gene turns red blood cells into rigid crescent or sickle shapes instead of soft discs. These misshapen cells get stuck in veins and arteries, blocking the flow of blood that carries life-giving oxygen to the body and causing the diseases horrifying hallmark: episodes of agony that begin in babyhood.

Millions of people globally, a vast majority of them Africans, suffer from sickle cell disease. Researchers have worked for decades on improving treatment and finding a cure, but experts said the effort has been hindered by chronic underfunding, in part because most of the estimated 100,000 people in the United States who have the disease are African American, often poor or of modest means.

The disease also affects people with southern European, Middle Eastern or Asian backgrounds, or those who are Hispanic, like Helen.

This is the story of two quests for a sickle cell cure one by the Obando family and one by a determined scientist at Boston Childrens Hospital, Dr. Stuart Orkin, 73, who has labored against the disease since he was a medical resident in the 1970s.

Like many others affected by sickle cell, the Obando family faced a double whammy: not one but two children with the disease, Helen and her older sister, Haylee Obando. They lived with one hope for a cure, a dangerous and sometimes fatal bone marrow transplant usually reserved for those with a healthy sibling as a match. But then they heard about a potential breakthrough: a complex procedure to flip a genetic switch so the body produces healthy blood.

Scientists have been experimenting with gene therapy for two decades, with mixed success. And it will be years before they know if this new procedure is effective in the long term. But if it is, sickle cell disease could be the first common genetic disorder to be cured by manipulating human DNA.

Four weeks after the infusion of stem cells, Helen was strong enough to be discharged. At home, in Lawrence, Massachusetts, on a sofa with her mother by her side, she put a hand over her eyes and started to sob. She and her family wondered: Would it work? Was her suffering really over?

A Familys Nightmare

Sheila Cintron, 35, and Byron Obando, 40, met when she was in the eighth grade and he was a high school senior. They fell in love. Haylee, their first child, was born in 2001, when Cintron was 17.

When a newborn screening test showed that Haylee had the disease, her father asked, Whats sickle cell?

They soon found out.

As the family gathered for her first birthday party, Haylee started screaming inconsolably. They rushed her to the hospital. It was the first of many pain crises.

Doctors warned the parents that if they had another baby, the odds were 1 in 4 that the child would have sickle cell, too. But they decided to take the chance.

Less than two years later, Helen was born. As bad as Haylees disease was, Helens was much worse. When she was 9 months old, a severe blockage of blood flow in her pelvis destroyed bone. At age 2, her spleen, which helps fight bacterial infections, became dangerously enlarged because of blocked blood flow. Doctors surgically removed the organ.

After Helen was born, her parents decided not to have any more children. But four years later, Cintron discovered she was pregnant again.

But they were lucky. Their third child, Ryan Obando, did not inherit the sickle cell mutation.

As Ryan grew up, Helens health worsened. When he was 9, Helens doctors suggested a drastic solution: If Ryan was a match for her, he might be able to cure her by giving her some of his bone marrow, though there would also be major risks for her, including death from severe infections or serious damage to organs if his immune system attacked her body.

As it turned out, Ryan matched not Helen but Haylee.

The transplant succeeded, but her parents asked themselves how they could stand by while one daughter was cured and the sicker one continued to suffer.

There was only one way to get a sibling donor for Helen: have another baby. In 2017, the couple embarked on another grueling medical journey.

Obando had a vasectomy, so doctors had to surgically extract his sperm from his testicles. Cintron had 75 eggs removed from her ovaries and fertilized with her husbands sperm. The result was more than 30 embryos.

Not a single embryo was both free of the sickle cell gene and a match for Helen.

So the family decided to move to Mesa, Arizona, from Lawrence, where the cold, which set off pain crises, kept Helen indoors all winter. The family had already sold their house when they heard that doctors at Boston Childrens were working on sickle cell gene therapy.

Cintron approached Dr. Erica Esrick, a principal investigator for the trial. But the trial wasnt yet open to children.

Figuring Out the Science

Nothing had prepared Orkin for the suffering he witnessed in his 30s as a medical resident in the pediatric hematology ward at Boston Childrens. It was the 1970s, and the beds were filled with children who had sickle cell crying in pain.

Orkin knew there was a solution to the puzzle of sickle cell, at least in theory: Fetuses make hemoglobin the oxygen-carrying molecules in blood cells with a different gene. Blood cells filled with fetal hemoglobin do not sickle. But the fetal gene is turned off after a baby is born, and an adult hemoglobin gene takes over. If the adult gene is mutated, red cells sickle.

Researchers had to figure out how to switch hemoglobin production to the fetal form. No one knew how to do that.

Orkin needed ideas. Supported by the National Institutes of Health and Howard Hughes Medical Institute, he kept looking.

The breakthrough came in 2008. The cost of gene sequencing was plummeting, and scientists were finding millions of genetic signposts on human DNA, allowing them to home in on small genetic differences among individuals. Researchers started doing large-scale DNA scans of populations, looking for tiny but significant changes in genes. They asked: Was there a molecular switch that flipped cells from making fetal to adult hemoglobin? And if there was, could the switch be flipped back?

They found a promising lead: an unprepossessing gene called BCL11A.

In a lab experiment, researchers blocked this gene and discovered that the blood cells in petri dishes started making fetal instead of adult hemoglobin.

Next they tried blocking the gene in mice genetically engineered to have human hemoglobin and sickle cell disease. Again, it worked.

Patients came next, in the gene therapy trial at Boston Childrens that began in 2018.

The trial run by Dr. David Williams, an expert in the biology of blood-forming stem cells at Boston Childrens, and Esrick has a straightforward goal: Were going to reeducate the blood cells and make them think they are still in the fetus, Williams said.

Doctors gave adult patients a drug that loosened stem cells immature cells that can turn into red blood cells from the bone marrow, their normal home, so they floated free in the bloodstream. Then they extracted those stem cells from whole blood drawn from the patient.

The researchers used a disabled genetically engineered AIDS virus to carry information into the stem cells, flipping on the fetal hemoglobin gene and turning off the adult gene. Then they infused the treated stem cells into patients veins. From there, the treated cells migrated into the patients bone marrow, where they began making healthy blood cells.

With the success in adults, the Food and Drug Administration said Boston Childrens could move on to teenagers.

When her mother told her about the gene therapy trial, Helen was frightened. But the more she thought about it, the more she was ready to take the risk.

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared.

Helen was scheduled for her six-month checkup Dec. 16. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved, even with blood transfusions. She had no signs of sickle cell disease.

More:
At 16, shes a pioneer in the fight to cure sickle cell disease at Boston Childrens - Boston.com

Read More...

Novartis opens facility for innovative cell and gene therapies in Switzerland – Science Business

Sunday, January 12th, 2020

In the presence of Federal Councillor Alain Berset and other distinguished guests, Novartis inaugurated a new manufacturing facility for cell and gene therapies at Stein, Switzerland on November 28th.

Our site in Stein is vital for new launches of solid and liquid drugs, said Steffen Lang, Global Head of Novartis Technical Operations and member of the Novartis Executive Committee. "The construction of the new manufacturing facility is another investment in the production of breakthrough cell-based therapies that can potentially change the lives of patients.

In addition to manufacturing areas for novel CAR-T cell therapies, the new building also hosts the production of innovative, difficult-to-manufacture solid dosage forms such as tablets and capsules. In September 2019, the first clinical production of a cell and gene therapy batchwas successfully completed.

Unlike conventional drug production, cell and gene therapy asks for the manufacture of a personal dose for each patient. For this purpose, patients who have already undergone various therapies have a small amount of their own blood cells taken, which are then sent to Stein. "Here we enrich part of the white blood cells, the T cells, and genetically modify them so that they can recognize and fight the cancer cells in the patient's blood," says Dorothea Ledergerber, project manager of the Stein plant for cell and gene therapies. The altered cells are then sent back to hospital and administered to the patient by infusion. Novartis is doing pioneering work here: "We have the unique opportunity to offer patients for whom there have been no other therapeutic options a totally new perspective by using these novel CAR-T cell therapies," says Dorothea Ledergerber.

Read more in German

This release wasfirst publishedby Novartis.

Read more:
Novartis opens facility for innovative cell and gene therapies in Switzerland - Science Business

Read More...

Page 43«..1020..42434445..5060..»


2025 © StemCell Therapy is proudly powered by WordPress
Entries (RSS) Comments (RSS) | Violinesth by Patrick