StemCell Therapy

INCHEON AIRPORT, SEOUL, SOUTH KOREA - 2018/06/12: A young girl communicating with a robot that is on ... [+] display at Incheon International Airport in Seoul / South Korea. The Guide Robot recognises languages; In addition to English, it can also answer Korean, Chinese and Japanese and recognise boarding cards that are scanned on the touch screen. In South Korea robots are playing an increasing role in society. AI boosts the economy in the countrys rapidly growing industry. Still, threats by terminators and lost employment threats linger. (Photo by Jonas Gratzer/LightRocket via Getty Images)

The world of social technology and public-private partnerships has been rapidly changing, and organizations must adapt their strategies to stay ahead of the curve. UN recently announced that the Republic of Korea is one of the top leaders in the 2022 digital government ranking of the 193 United Nations Member States, scoring the highest in the scope and quality of online services, the status of telecommunication infrastructure, and existing human capacity. LG NOVA is a perfect example of a private sector enterprise constantly innovating and looking for new ways to drive growth. LG NOVA is a subsidiary of LG Electronics, one of the world's largest electronics firms headquartered in Korea. It is on a critical mission to reinvent social impact for the future. The company has been at the forefront of innovation for over five decades, and its commitment to social responsibility and sustainability has earned it a reputation as a trusted partner for businesses and organizations around the globe. In fact, LG Energy Solutions is teaming up with Honda to build the $3.5 billion battery factory and create 2,200 jobs, with an overall investment projected to reach $4.4 billion as part of a joint venture in southern Ohio. President Joe Biden recently mentioned that the Honda-LG joint venture was "committing more than $5 billion toward electric vehicle battery manufacturing and factory retooling across the state.

To realize its vision of becoming a leading global provider of social technology solutions, LG NOVA has invested heavily in its Mission for the Future program. The program is designed to identify and invest in early-stage companies working on cutting-edge technologies with the potential to impact society positively.

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In Silicon Valley and globally, LG NOVA seeks to achieve a leadership position in socially impactful technology solutions. The company is focused on investing in early-stage companies that are working on cutting-edge technologies with the potential to impact society positively. Social innovation is reinventing how we live, work, and play, and LG NOVA is committed to supporting the companies leading this charge. The organization can drive growth and create value for shareholders, customers, and partners.

According to Dr. Sokwoo Rhee, Corporate SVP of Innovation at LG Electronics and Head of the North America Innovation Center at LG NOVA, The key industries that the LG NOVA program is seeking submissions from are digital health, electric mobility, and the metaverse. Digital Health is going to be huge. Healthcare is already huge, and you can see that many people are jumping into it right now. Electric mobility is also exploding with new solutions for electric vehicles and charging infrastructure. The government is putting money into the market, so it will only get bigger. While metaverse is still in its early stage of development, LG is continuing to explore how we can play a leading role in its development. Its a key part of our future, and we anticipate that at some point, we'll have an excellent idea or solution that we can make commercially viable."

Dr. Sokwoo Rhee, Corporate SVP of Innovation at LG Electronics and Head of the North America ... [+]ca Innovation Center at LG NOVA speaking at 2022 LG NOVA Innovation Festival

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Significant trends in healthcare and electric mobility include the convergence of physical and digital health, the rise of personalized and precision medicine, the growth of the wearables market, and the increasing demand for EV infrastructure as the market for EVs grows. In the metaverse, the initiative looks for applications that can create new value for users by providing an immersive and interactive experience. Dr.Rhee, previously an Associate Director for Cyber-Physical Systems (CPS) Innovation at the National Institute of Standards and Technology, U.S. Department of Commerce, continued to emphasize that "In my opinion, healthcare is a key part of smart communities. Now, if you have to go to a hospital, it's not exactly a smart city; it's just a hospital. But when you are doing healthcare at home, also known as digital healthcare, it broadens to become a community issue. So I believe digital health indirectly contributes to the concept of a smart city." The notion of the smart city is a significant trend to watch in the industry, as it represents the future of urban living and has the potential to create substantial value for companies that can provide solutions that make cities more efficient and livable.

Moreover, according to Joshua Di Frances, Head of Incubation and Senior Director, Healthcare Strategy & Enterprise Business Development, "I focus primarily on healthcare and digital health. Some companies are hospital- and consumer-facing, but they're all looking at the patient and trying to improve health. But I think some companies in that area, like Digbi Health, are exciting. They're looking at personalized health using DNA, genetics, and microbiome to understand better what each person needs. Personalizing medicine represents a huge area of growth. Medicine is going that way; we've seen it for several years. LG has a screen presence at hospitals and a hardware presence, but we also have a powerful consumer brand, and healthcare is moving to the home. And I think there's an opportunity when you think about connected devices, remote patient monitoring, and empowering patients to understand their health better where we could play a significant role. I think our leadership sees that, and thats why health is a big area of focus for us."

According to Maria Patterson, Director of New Ventures, LG Electronics, and LG NOVA Entrepreneur in Residence, Electric Vehicles are a priority in the near future due to their lack of emissions and ability to be powered by renewable energy sources. However, there are concerns about the potential for EVs to overload the electrical grid, as they require a significant amount of power to charge. One way to address this issue is by using AI to model people's behavior and better understand when and how they will use their EVs. This information can then be used to manage the charging of EVs in a way that does not overload the grid. Additionally, it is essential to ensure that these new technologies are accessible to everyone. Those who cannot afford EV chargers should not have to pay for infrastructure improvements that primarily benefit those who can. She states, "Using AI to model peoples behavior is very interesting because it makes us realize everything is very much interconnected, especially with electrification.

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Based on the above information, it is evident that LG Electronics is focusing on emerging industries to stay ahead of the curve and create value for their shareholders, such as healthcare and electric vehicles. This aligns with vital global megatrends. The need for better healthcare solutions will only increase as the world's population ages, and the move towards electric vehicles is gathering momentum to combat climate change. LG is well-positioned to capitalize on these trends and create significant value for its shareholders in the years to come.

2022 LG NOVA Innovation Festival

LG NOVA has spotlighted several attractive solutions focusing on health care and electric vehicles as part of their leading finalists for the Mission for the Future challenge.

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With digital health capabilities key to delivering healthcare to diverse and disparate communities, LG NOVA is working on expanding and scaling greater access to healthcare and personal health services.

Digbi Health is a digital care platform to prevent and reverse chronic metabolic, digestive, and mental health conditions using gut microbiome insight, genetics, AI, and food-as-medicine. Together LG and Digbi will work to make care globally accessible at home and the office and advance cure.

LifeNome is a B2B2C precision health platform powered by genomics and AI, offering personalized health and well-being solutions to the world's leading enterprises. LG and LifeNome plan to bring to market the world's first precision maternal and family health platform, supporting pregnant individuals 24/7 from conception to the early stages of a child's life.

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Mindset Medical is a sensor-based technology platform that can use a camera in patient-owned devices to capture health and biological readings to assist doctors in enhancing medical diagnosis and treatment effectiveness. Expanding upon new services available to doctors and providers, LG's work with Mindset Medical is expected to make it easier for medical providers to understand patient health over time or during live virtual doctor visits.

XRHealth develops virtual treatment rooms, integrating immersive VR/AR technology, licensed clinicians, and real-time data analytics on one platform, providing a comprehensive therapeutic care solution for patients through the continuum of care, from the hospital to the patient's home. LG and XRHealth will continue to grow the service offering to deliver more insights to clinicians and enable more precise personalized care.

The solutions in this category lead to social innovation by bringing technology-enabled solutions to healthcare's most challenging problems and making it easier for people to get the care they need. Specifically, these companies are focused on making it easier for people to access care, whether that's through at-home solutions or by providing more data to doctors to help them make better decisions.

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With LG's commitment to sustainability as an organization, LG NOVA is working to pursue new opportunities to expand access to electric vehicles (EV) and electric mobility solutions by enabling more businesses to leverage their infrastructure to provide EV services.

Driivz empowers major EV service providers with an end-to-end EV charging and intelligent energy management software platform. LG and Driivz will work together to enable the hospitality industry across the United States to offer EV charging-as-a-service while optimizing their EV charging operations and providing their customers with an exceptional EV charging experience.

I-EMS Solutions, Ltd. uses innovative AI and blockchain-based distributed energy resource management systems (DERMS) and transactive energy software platforms to enable power optimization across smart cities, smart homes, and e-mobility. By working with I-EMS, LG will help modernize the electricity grid and optimize energy management to deliver better power management solutions for the market.

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SparkCharge offers an affordable and convenient way for electric vehicle owners to charge their EVs without a direct-access charger at home or on the road. LG and SparkCharge plan to work together to deliver turnkey EV solutions for businesses to create a stream of revenue from their parking spaces.

The companies in this category focus on making it easier for people to switch to electric vehicles and expanding the infrastructure necessary to support widespread adoption. This is an integral part of LG's commitment to sustainability, as electric vehicles have a lower environmental impact than traditional gas-powered cars.

LG NOVA's Mission for the Future program is a testament to the pressing need for social innovation globally. The company is working with startups in various industries to create new solutions for some of the world's most pressing problems. LG NOVA is committed to positive change in the world, from healthcare to electric vehicles. Other organizations can begin to emulate this program by establishing social innovation initiatives and partnering with startups to create new technologies and services that address pressing global issues.

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Important Mission By LG To Reinvent Society With Future Growth - Forbes

TUCSON, Ariz., Oct. 13, 2022 /PRNewswire/ -- Learn Look Locate (LLL) announced a partnership with Myriad Genetics, a leader in genetic testing and precision medicine, to advance awareness about hereditary breast cancer and risk assessment. As part of the partnership, Myriad will share educational information for breast cancer patients and survivors on LLL's doctor monitored and curated platform.

"We are partnering with Learn Look Locate to help further educate patients and healthcare providers about hereditary cancer testing," said Thomas Slavin, M.D., chief medical officer, Myriad Genetics. "This testing provides vital data-driven genetic insights and personalized medical information to help people with a personal or family history of cancer understand their risk of being born with a predisposition to cancer. With this information, patients and their providers can take steps to either proactively prevent disease or, if diagnosed with cancer, personalize their treatment."

The American Society of Breast Surgeons (ASBrS) recommends testing of all patients diagnosed with breast cancer. Patients without a breast cancer diagnosis, but have a family history of the disease, should discuss testing with their physicians. Genetic testing can provide an assessment of a patient's risk of developing breast cancer and provide data-driven medical information for use in personalizing medical management plans.

Myriad's MyRiskHereditary Cancer Test offers multi-gene panel testing that helps determine a patient's hereditary cancer risk associated with 11 primary cancer types, including breast cancer. Patients without breast cancer may also be eligible for further personalized risk stratification with RiskScore, a risk assessment tool that delivers a personalized five-year and remaining lifetime risk of breast cancer

"Genetic testing is one of the most powerful pieces of knowledge about yourself," said Allison DiPasquale, M.D., Breast Surgical Oncologist at Texas Oncology. "It can help predict your risk of certain cancers, help guide systemic therapy and surgical recommendations, as well as provide potential lifesaving information for relatives. Learning how to tap into the insights of your genes empowers both patients and family members. I am so honored to be part of LLL and partner with Myriad on the LLL webpage."

To learn more about hereditary cancer testing, visit LLL online. And stay up to date on the latest therapies and breast cancer news by following LLL on social media @learnlooklocate Facebook, Instagram, Twitter, LinkedIn.

About Myriad Genetics

Myriad Genetics is a leading genetic testing and precision medicine company dedicated to advancing health and well-being for all. Myriad develops and offers genetic tests that help assess the risk of developing disease or disease progression and guide treatment decisions across medical specialties where genetic insights can significantly improve patient care and lower healthcare costs. Fast Company named Myriad among the World's Most Innovative Companies for 2022. For more information, visit http://www.myriad.com.

About Learn Look Locate (LLL)

Founded in 2019 by breast cancer survivor, Cynthia Jordan, LLL represents a global movement for breast cancer support and education. As part of her guiding mission to diagnose breast cancer early and educate people, LLL provides answers to the most important questions patients have: What do I need to know? Who are my resources? Where do I turn now?

LLL focuses on early detection and early diagnosis, aggregating the latest technology and research, the most cutting-edge breast cancer treatments, and information from globally recognized doctors/specialists. Understanding breast cancer at the earliest possible point of a patient's journey is critical. LLL empowers everyone touched by this diagnosis with a supportive community who share their breast cancer stories all ages, genders, and at all stages of the disease.

Join the global movement and become part of the conversation at: http://www.LearnLookLocate.com.

Media Contact:Cynthia Jordan1-407-592-4474[emailprotected]

SOURCE Learn Look Locate

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Learn Look Locate Partners with Genetic Testing Leader, Myriad Genetics, in Educational Campaign - PR Newswire

If youve ever lied about your height on a dating app, we now know to whom you can address your grievances. No, not any reinforced societal expectations about what heterosexual relationships look like or how traditional masculinity or femininity manifests, but a stretch of genetic variations.

Height is one of the easiest physical characteristics to measure, and we know that its a trait with high hereditability, meaning inherited genes almost entirely determine it. This trait seems like it should be a fairly straightforward one to parse genetically, but the actual places where gene variations occur have been hard to pin down.

Researchers in Australia, the U.K., and the U.S. published a study on Wednesday in the journal Nature that provides evidence for more than 12,000 spots in the human genome that are associated with height.

Whats new In the largest and most diverse genome-wide association study (GWAS) to date, the studys authors have identified 12,111 places in the human genome associated with height. The team analyzed existing genetic data from nearly 5.4 million people, more than a million of whom were not of European ancestry.

Throughout ones DNA are single nucleotide polymorphisms (SNP, pronounced snip), which are spots for genetic variation. SNPs are, in part, whats responsible for eye and hair color, height variation, and pretty much any other inherited trait. These arent mutations but spots where a particular nucleotide (adenine, cytosine, guanine, and thymine) exists. These nucleotides encode certain traits.

This study has pinpointed more than 12,000 locations in the human genome where those SNPs coding for height occur.

Why it matters As simple as height may seem, theres so much we still dont know about it.

Loic Yengo, lead author, and statistical geneticist at the University of Queensland, says this work sets the stage for future genetic investigation of other traits and diseases, demonstrating that genetic data alone can contribute a significant portion of trait variation and risk of disease.

It also offers a guide on sample sizes. Though this is the largest GWAS to date, it will likely impel broader and broader studies with even more people.

Widening the sample size is important for the future of personalized medicine, Yengo wrote in an email to Inverse. The more genetic material included, the better researchers will get at identifying at-risk patients before an inherited disease or trait progresses beyond prevention.

More directly, this study teaches something about the biology of bone growth and can eventually help us design treatments for certain skeletal growth disorders, Yengo wrote to Inverse.

Digging into the details Genetic information on European ancestry is now saturated. There arent many more unknown common variants associated with height in this population. According to Yengo, these more than 12,000 genetic variants account for about 40 percent of height variation in European ancestry.

Weve more or less finished mapping them, at least for European ancestry, Joel Hirschhorn, senior author, and a pediatric endocrinologist at Boston Childrens Hospital tells Inverse. Genetic rarities, however, still remain. Yengo now wants to focus on rare variants present in less than 1 percent of that population.

And, there are plenty of other ancestry groups that must be investigated for common variants. In this study, fewer than 78,000 people represented South Asian ancestry. But Hirschhorn and Yengo point to the importance of better understanding height in African ancestries.

Ancestries with an s because of the large diversity existing on the African continent, Yengo writes. Considering human life originated in Africa, information from these populations could provide novel insights.

Humans all started as one relatively small group in Africa, Hirschhorn says. Some of that genetic diversity left Africa, and some of it stayed behind. Since these genes come from much older populations, it has more genetic variability because its had so much more time to evolve.

Theres more to discover and better ability to pinpoint where the right variants are, but to do that, you need large studies of folks with African ancestry, he says.

Whats next These analyses will only get bigger and more diverse. Yengos vision for this researchs future is to home in on rarer genetic variants that alter height and to find even more height variants in other populations.

Hirschhorns interested in the immediate biological understanding we can glean. The more relevant genes we understand, the better we can get to know the biological mechanisms behind how height and skeletal growth work. He also says this study offers an opportunity for prevention and intervention when it comes to genetic disorders or diseases that affect height.

And as a pediatric endocrinologist, I see a lot of kids where the parents come in because theyre concerned about their child growing too slowly, he says. As sci-fi as it may seem, its not too hard to look at that kids genetics to see if perhaps theyre going to end up on the shorter side based on variants identified. But if that kids genetics suggest they should end up on the taller side, then there may be something else going on worth investigating.

Nobody needs genetic testing to at least partially, if not mostly, understand their own height. If youre curious, see if you can learn your parents heights, and that should offer some insight.

LEARN SOMETHING NEW EVERY DAY.

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Scientists identify more than 12,000 spots in the human genome associated with height - Inverse

Former chairman of CareDx, Inc. and seasoned global industry executive with track record of transforming organizations will lead Kyverna and drive its growth as it aims to bring a new class of targeted gene therapies to treat severe immune-related diseases

Company plans to submit Investigational New Drug application for its lead program KYV-101, a novel CD19 CAR T-cell therapy, for the treatment of lupus nephritis

Dominic Borie, M.D., Ph.D., named president, research and development to continue advancing company's core program KYV-101 and strategic partnerships with Gilead and Intellia

Kyverna's therapeutic platform combines advanced T-cell engineering and synthetic biology technologies to suppress and eliminate autoreactive immune cells at the root cause of inflammatory disease

EMERYVILLE, Calif., Oct. 13, 2022 /PRNewswire/ -- Kyverna Therapeutics ("Kyverna"), a cell therapy company with the mission of engineering a new class of therapies for serious autoimmune diseases, today announced the appointment of Peter K. Maag, Ph.D., as the company's chief executive officer (CEO). Dr. Maag succeeds Dominic Borie, M.D., Ph.D., who has been named Kyverna's president of research and development (R&D). Dr. Maag also joins Kyverna's board of directors.

"We are thrilled to welcome Peter as Kyverna's new CEO," said Ian Clark, chair of Kyverna's board of directors. "Peter is an accomplished executive with extensive industry experience at private and public companies spanning three continents.His consistent record of driving transformational growth in healthcare through product innovation, creative business development, sequential financings, and sound investment strategieswill be invaluable to progress Kyverna and deliver on its mission to help bring new and much-needed treatment options to patients with serious autoimmune and inflammatory diseases."

Dr. Maag has more than 20 years of executive management experience in the pharmaceutical and diagnostic industry. Most recently, he was executive chairman, president and CEO of CareDx where he had built the company from a small start-up into a public company and industry-leading powerhouse in transplantation through a series of BD&L and financing transactions. CareDx serves the transplant community with breakthrough technologies in more than 60 countries and has created a strong presence in this specialty market. Prior to joining CareDx in 2012, Peter held multiple positions in Novartis with increasing responsibilities. As President of Novartis Diagnostics, he drove growth and innovation in its blood-screening business. Previously, he led one of Novartis' key affiliates as country president, Germany, and lived in a dynamically growing market as country president, Korea. At headquarters in Switzerland, he served as the head of strategy for Novartis Pharmaceuticals and helped launch the Infectious Diseases franchise.

"I am passionate about patient care and am honored to be appointed to lead Kyverna and this extraordinarily talented team. This is such an exciting time to join the company with key milestones like our submission of an IND for our lead candidate, KYV-101 in lupus nephritis tomorrow. This important regulatory milestone will provide a runway to move KYV-101 into the clinical phase and brings us a step closer to developing a new treatment option for lupus nephritis," said Dr. Maag. "I believe that Kyverna is at the forefront of revolutionizing how we treat severe immune-related and inflammatory diseases with its new class of cell therapies. With multiple shots on goal, I believe that Kyverna is well positioned to transform how autoimmune diseases are treated."

Prior to joining Novartis, Peter worked at McKinsey & Company in New Jersey and Germany, focusing on pharmaceuticals and globalization strategies. Besides supporting various healthcare and tech companies in their growth efforts, he holds board and advisory positions at Phoenix Pharma SE, CareDx, MiroMatrix, and the Personalized Medicine Coalition. Peter studied pharmaceutical sciences in Heidelberg, London, and Berlin.

In addition to Dr. Maag's appointment, Kyverna announced that Dr. Borie will step away from his CEO role and has been named president of R&D.

"We are grateful for the significant contributions Dominic has made in bringing Kyverna out of stealth in 2020 and growing the company and building out Kyverna's core programs," added Mr. Clark. "Dominic has performed admirably, and we could not be more pleased that he will serve as president of Kyverna's R&D operations. We look forward to working closely with him and his team to continue moving forward our autologous and allogeneic programs, as well as our collaborations with Gilead and Intellia so that we can continue to strive to bring new treatments to patients rapidly."

About KYV-101KYV-101 is an autologous version of a novel clinical-stage anti-CD19 chimeric antigen receptor T-cell (CAR T) construct with properties well suited for use in B cell-driven autoimmune diseases such as lupus nephritis, systemic sclerosis, and inflammatory myopathies. Kyverna has obtained exclusive, worldwide licenses from the National Institutes of Health (NIH) to use this CD19 construct in both autologous and allogeneic CAR T-cell therapies.

About Kyverna TherapeuticsKyverna Therapeutics is a cell therapy company with the mission of engineering a new class of therapies for autoimmune and inflammatory diseases. The Kyverna therapeutic platform combines advanced T-cell engineering and synthetic biology technologies to suppress and eliminate the autoreactive immune cells at the origin of autoimmune and inflammatory diseases. In addition to aiming to develop next-generationchimeric antigen receptor T-cell (CAR T) therapies in both autologous and allogeneic settings, Kyverna is creating synReg T cells, a synthetic version of Regulatory T cells (Tregs), powerful natural immune cells that control immune homeostasis through multiple immunosuppressive mechanisms. By offering more than one mechanism for taming autoimmunity, Kyverna is positioned to act on its mission of transforming how autoimmune diseases are treated. For more information, please visit https://kyvernatx.com.

SOURCE Kyverna Therapeutics

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Kyverna Therapeutics Names Peter Maag, Ph.D., as Chief Executive Officer - PR Newswire

Pokagon Flag

Harper Institute collecting Native American samples to address health disparities

Dr. Gerald Morris, the medical director for the Pokagon Band of Potawatomi Indians that live in northern Indiana and southwest Michigan, isnt certain that the Native American patients he sees show higher rates of cancer than the rest of the population though he suspects it.

The one thing that stands out to me is, oftentimes, theyre more advanced when we find them, said Morris, who is a Pokagon citizen. I think they have the same issues as other patients in terms of the types of cancers. Its just that they tend to be more advanced when I do see them.

Morris said the reason the cancers are more advanced could trace back to historical access to health care, which leads to lower rates of cancer screening. He hopes that a partnership being developed between the Pokagon Band and the University of Notre Dame'sHarper Cancer Research Institute (HCRI) will help raise awareness of the importance of screening and early detection to address measurable disparities in cancer treatment outcomes involving Native Americans.

The Pokagon Bands tribal leadership plans to invest $25,000 to help fund the project. Scott Brewer, a senior vice president at the tribes Four Winds Casino Resorts, oversees the companys philanthropic activities and proposed the HCRI project to his tribal council. He said the research disparities Harper presented speak for themselves of about 90,000 cancer tissue samples in the Cancer Genome Atlas at the National Cancer Institute, only 114 came from Native Americans.

Thats .001 percent of the total, even though people identifying as at least partially American Indian or Alaska Native constitute about 3 percent of U.S. population.

Weve partnered with the University on several things, but this is the first health initiative, Brewer said. It was the easiest sell for me because its research to try to get a cure, and the research is specific to Native Americans.

The project aims to double the number of cancer tissue samples that come from Native Americans through several outreach efforts. While Harper is in the early stages of relationship building with the Pokagon Band, it has been working for a few years with other minority groups: the Kalispel tribe in eastern Washington state, and medical groups in Puebla, Mexico.

Andy Bullock, associate director of HCRI, explained why its so important to diversify the cancer tissue samples available for research. His rationale starts with the direction the future of cancer research is taking.

Cancer is not one disease, its thousands, Bullock said. It used to be cancer of the breast, colon, kidney, etc. With more personalized medicine, its now more about the type of mutation.

Cancer comes from a mutation in a persons DNA, leading cells to grow uncontrollably in different ways. Targeted cancer drugs and chemotherapy attempt to block different pathways that are turned on by these mutations in the body. But people of different race or ethnicity with the same cancer might have different mutations that activate the cancer-driving pathway.

It might be different between a Caucasian person and Native American person and African American person, said M. Sharon Stack, the Anne F. Dunne and Elizabeth Riley Director of HCRI. We don't know for most cancers in minority populations because nobodys really looked.

Most cancer tissue is collected at elite cancer centers, where there is an over-representation of white patients that can afford treatment there. Minority cancer patients are more likely to seek treatment at local, often less-costly cancer centers. These trends drive the disparity that leads to such low numbers of Native Americans in the Cancer Genome Atlas.

The current model is we come up with a drug that helps white people, then hope it helps other people too, Bullock said. We are trying to invert that model. We want to research Native Americans and see how well those pathway blockers help them and other people.

Stack said the project fits the Universitys mission of serving populations often marginalized in the past, but its also a really compelling scientific question.

A 2022 American Cancer Society report on American Indian and Native Alaskan populations found that cancer incidence in these indigenous communities is higher nationally than among whites, especially for lung, colorectal and kidney cancers. The report notes that lower levels of health insurance and chronic underfunding of the U.S. Indian Health Services exacerbates the problem.

Within the national numbers are wide variations, partly because there are 574 federally recognized tribes and more than 200 that remain unrecognized. Even when factors like health care and poor housing are equalized, wide disparities in cancer cases remain. For instance, Northern Plains Indians are four times more likely to have kidney cancer than whites. Survivability charts also show disparities, likely due to later detection.

Bullock said HCRI has been working for several years with partners in eastern Washington because an alumnus there donated money for cancer research. Ryan Gee, who graduated in 1998, is CEO of Gee Automotive companies with 35 car dealerships across several western states.

Gee, whose wife survived cancer, said the fight against the disease is his side passion. He co-founded the Community Cancer Fund with a friend who survived cancer against great odds. The organization raises money for cancer research and to help families in the Northwest cope with the severe economic impact of cancer treatment.

The Kalispel Indian tribe is a large donor to the fund, so Gee asked Harper about cancer research among Native Americans and learned it was lacking. What if we set a goal to double the amount of Native American research samples in the database through Harper at Notre Dame, Gee said.

Gees nonprofit works with the MultiCare hospital system in Washington state, which has helped Harper collect about 20 cancer tissue samples from Native Americans so far.

Native American populations experience much higher cancer rates than non-Hispanic white people in the U.S and are historically under-represented and critically under researched, said Annie Reedy, MultiCares chief research and education officer. Through this collaboration with Notre Dame we are moving toward a greater understanding of cancers in Native Americans and building a foundation for future clinical trials and treatment advances for this population.

Fundraising is an important part of this research because the cancer tissues are expensive to analyze. When a lump or tumor is removed from a patient, the doctors will perform a biopsy on a small slice to determine if its cancerous. The rest is considered medical waste.

Stack said it can be difficult to convince people to share the leftover portions with tissue banks, especially if the target population is mistrustful of the medical community. Relationship building can take years. A tissue bank must strip out identifying information that could lead back to the donor, leaving only basic demographic information.

Harper contracts with a Chicago company that extracts DNA from samples and looks for about 600 mutations linked to cancer. The $2,500 process also analyzes the RNA sequence, which is a blueprint of the DNAs genetic code that gets made into the proteins that form hair and skin and bones the different parts of the body. Mens basketball coach Mike Brey raised money for the project through his Coaches vs. Cancer work.

Another branch of this ongoing project works with partners in Puebla, Mexico. There is a huge disparity in childhood leukemia survival between people in America and Mexico. In America, more than 90 percent of leukemia patients live to the 10-year marker that signals successful treatment. In Mexico, only about 60 percent live for four years.

HCRI has been collecting samples from pediatric patients in South Bend to compare them with samples from Mexican patients.

Ideally we would have Caucasian kids and also kids of Latin American origin that happened to be raised in South Bend to see if any differences are genetic, or if it could be an environmental effect, Stack said.

Maria Cristina Miranda Vergara earned a doctoral degree in biochemistry from Notre Dame in May. As a student, she studied biomarkers that could improve early cancer detection, working with partners at the Catholic Universidad Popular Autnoma del Estado de Puebla. Now working in biochemical research in Puebla, Miranda Vergara helps process Mexican samples using the same protocols so that the data can be compared to Notre Dames research.

My dream is to develop some sort of microfluidic device that would allow us to detect cancer biomarkers in the field, so we could do early diagnosis of children in their communities, she said. Its difficult to determine the symptoms of cancer, but we need to improve early detection to get the right treatment and improve outcomes.

The common thread between the Harper projects in Mexican and Native American communities is to improve cancer survival by analyzing distinct populations that have not been studied as thoroughly in the past.

Priscilla Gatties, director of Health Services for the Pokagon Band, said Harpers research can help address disadvantages that Native American communities have developed due to a lack of historical health data. She said her mother-in-law, a Pokagon citizen, is fighting against kidney cancer using medication that didnt exist a decade ago.

From my perspective, Im very excited about what Harper is doing, Gatties said. Theres a lot of new research and information, and we can be a part of that. I think thats going to help patients and save lives, even if its later on down the road.

Read more from the original source:
The future of cancer research | News - ND Newswire

Future Market Insights Global and Consulting Pvt. Ltd.

The laboratory information systems market in Germany is set to exhibit a growth at a 9.9% CAGR during the forecast period 2022 -2030. Sales of laboratory information systems in China are forecast to increase at a 9.4% CAGR over the forecast period

NEWARK, Del, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The global laboratory information systems market is worth US$ 1.9 Bn as of now and expected to reach US$ 4.2 Bn by the year 2030 at a CAGR of 10.2% between 2022 and 2030.

Laboratory Information System (LIS) comes across as a collection of operating systems, software, and hardware used for processing, storing, and managing data in the clinical laboratories. As such, it has turned out to be one of the essential tools to support operations in the modern laboratories. They are inclusive of managing and processing data pertaining to several processes and testing.

The healthcare vertical has witnessed several technological advancements in the last few years, and laboratory information systems are no exception. The application areas include sample tracking and also as enterprise resource planning tools managing several facets of informatics. The systems actually let end-users accumulate patients data and also record it on database for managing the patients better.

Besides, quick adoption of explicit research on genomic studies and automation is catalysing utilization of laboratory information systems. With growing biobanks, notable investments in the novel technologies, easily available laboratory information systems products, services, and stern regulatory requirements across all the industries, the laboratory information systems market is all set to grow on an unstoppable note in the near future.

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Additionally, standardization of clinical trials coupled with modernization of regulatory pathways in the Peoples Republic of China is aiding the growth of laboratory information systems market. Cloud-based services are being rapidly adopted. High-speed internet is another factor to be attributed to.

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The ongoing trend implies tabling dedicated laboratory information systems with custom, commercial, and open source software interface with diverse budgetary specifications. Future Market Insights has etched these findings with future perspectives in its latest market study entitled Laboratory Information Systems Market.

Key Takeaways from Laboratory Information Systems Market

North America holds the largest market share due to the US being subject to quick adoption of well-structured healthcare IT sector and lab automation and the status quo is expected to remain unchanged even in the forecast period.

Europe stands second, with Germany leading from the front. It is expected to witness a CAGR of 9.9% in laboratory information systems market in the forecast period due to rising adoption of automation.

The Asia-Pacific is expected to grow at the fastest pace in the laboratory information systems market with China yielding personalized medicine with feasible support from the government.

Cloud-based laboratory information systems are dominating the market and would continue to do so even going forward.

By application, clinics hold the largest market share (over 48%).

Automation, in line with Industry 4.0, is expected to accelerate the laboratory information system (LIS) market in the forecast period, says an analyst from Future Market Insights.

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Competitive Arena

CTI, in August 2021, entered into partnership with LabWare Inc. with the objective of expanding the formers laboratory services for supporting research related to cell & gene therapy.

Oracle Corporation, in December 2021, partnered with Cerner Corporation for jointly announcing acquisition of the latter.

Orchard Software Corporation, in May 2021, completed acquisition of Corwen, LLC for expanding molecular testing capabilities and make way for integrated and comprehensive LIS solutions.

PathWest, in January 2021, integrated its SoftBank (new LIS transfusion medicine module) with NBAs (National Blood Authoritys) BloodNet LIS Interface.

CompuGroup, in December 2020, completed acquisition of Schuyler House for strengthening the footprint all across the US.

LabVantage Solutions, in July 2020, did launch its 100% integrated Scientific Data Management Systems (SDMS) via its LabVantage 8.5 edition.

Abbott, in July 2019, came up with STARLIMS Life Sciences Solution 11.1 for managing complex testing and sample workflows.

What does the Report Cover?

Future Market Insights offers an exclusive perspective and various real-time insights on the laboratory information systems market in its latest study, presenting historical demand assessment of 2016 2021 and projections for 2022 2030.

The research study is based on component (hardware, software, and services), by delivery mode (on-premise and cloud-based), and by end-user (hospitals, clinics, independent laboratories, and likewise).

Browse Full Report @ https://www.futuremarketinsights.com/reports/laboratory-information-systems-market

Key Market Segments Covered in Laboratory Information Systems Industry Research

By Components:

By Delivery Mode:

By End User:

Hospitals

Clinics

Independent Laboratories

Others

By Region:

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Table of Content

1. Executive Summary

1.1. Global Market Outlook

1.2. Analysis and Recommendations

2. Global Laboratory Information Systems Market Overview

2.1. Introduction

2.1.1. Global Laboratory Information Systems Market Definition

2.1.2. Global Laboratory Information Systems Market Taxonomy

2.2. Market Dynamics

2.2.1. Drivers

2.2.2. Restraints

2.2.3. Opportunity

2.3. Global Laboratory Information Systems Market Trends

2.4. Global Laboratory Information Systems (LIS) Market Demand (in Value in US$ Mn) Analysis 2015-2021 and Forecast, 20222030

2.5. Global Laboratory Information Systems Market Size (Value) Forecast

2.5.1. Y-o-Y Growth Projections

2.5.2. Absolute $ Opportunity

Read More TOC.

Have a Look at Related Research Reports of Healthcare

Radiology Information System RIS Market Size: The Radiology Information System market is projected to register a CAGR of 7.6% during the forecast period, up from US$ 809.8 million in 2020 to reach a valuation of US$ 1,315.6 million by 2026

Clinical Information System Market Sales: The clinical information system market is expected to record a CAGR of 8% during the forecast period, reaching a valuation of US$ 1560 Mn by 2027

Laboratory Filtration Devices Market Trends: Laboratory Filtration Devices Market - Global industry segment analysis, regional outlook, share, growth; laboratory filtration devices market forecast 2017 to 2027 by future market insights

Oncology Information Systems Market Share: The oncology information systems market is projected to be worth US$ 2.59 Bn by 2022 and is expected to grow to US$5.4 Bn by 2032, at a CAGR of 7.8% from 2022 to 2032

Clinical Decision Support Market Growth: The global clinical decision support market is likely to reach a US$ 2.2 billion valuation in 2022

About Future Market Insights, Inc.

Future Market Insights, Inc. is an ESOMAR-certified business consulting & market research firm, a member of the Greater New York Chamber of Commerce and is headquartered in Delaware, USA. A recipient of Clutch Leaders Award 2022 on account of high client score (4.9/5), we have been collaborating with global enterprises in their business transformation journey and helping them deliver on their business ambitions. 80% of the largest Forbes 1000 enterprises are our clients. We serve global clients across all leading & niche market segments across all major industries.

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Call for Accurate Automation in Healthcare Practices to drive the Laboratory Information Systems (LIS) Market | Future Market Insights, Inc. - Yahoo...

Dublin, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The "Stem Cell Manufacturing Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.

The global stem cell manufacturing market size reached US$ 11.2 Billion in 2021. Looking forward, the publisher expects the market to reach US$ 18.59 Billion by 2027, exhibiting a CAGR of 8.81% during 2021-2027.

Stem cells are undifferentiated or partially differentiated cells that make up the tissues and organs of animals and plants. They are commonly sourced from blood, bone marrow, umbilical cord, embryo, and placenta. Under the right body and laboratory conditions, stem cells can divide to form more cells, such as red blood cells (RBCs), platelets, and white blood cells, which generate specialized functions.

They are widely used for human disease modeling, drug discovery, development of cell therapies for untreatable diseases, gene therapy, and tissue engineering. Stem cells are cryopreserved to maintain their viability and minimize genetic change and are consequently used later to replace damaged organs and tissues and treat various diseases.

Stem Cell Manufacturing Market Trends:

The global market is primarily driven by the increasing venture capital (VC) investments in stem cell research due to the rising awareness about the therapeutic potency of stem cells. Apart from this, the widespread product utilization in effective disease management, personalized medicine, and genome testing applications are favoring the market growth. Additionally, the incorporation of three-dimensional (3D) printing and microfluidic technologies to reduce production time and lower cost by integrating multiple production steps into one device is providing an impetus to the market growth.

Furthermore, the increasing product utilization in the pharmaceutical industry for manufacturing hematopoietic stem cells (HSC)- and mesenchymal stem cells (MSC)-based drugs for treating tumors, leukemia, and lymphoma is acting as another growth-inducing factor.

Moreover, the increasing product application in research applications to produce new drugs that assist in improving functions and altering the progress of diseases is providing a considerable boost to the market. Other factors, including the increasing usage of the technique in tissue and organ replacement therapies, significant improvements in medical infrastructure, and the implementation of various government initiatives promoting public health, are anticipated to drive the market.

Key Players

Key Questions Answered in This Report:

Key Market Segmentation

Breakup by Product:

Breakup by Application:

Breakup by End User:

Breakup by Region:

Key Topics Covered:

1 Preface

2 Scope and Methodology

3 Executive Summary

4 Introduction

5 Global Stem Cell Manufacturing Market

6 Market Breakup by Product

7 Market Breakup by Application

8 Market Breakup by End User

9 Market Breakup by Region

10 SWOT Analysis

11 Value Chain Analysis

12 Porters Five Forces Analysis

13 Price Analysis

14 Competitive Landscape

For more information about this report visit https://www.researchandmarkets.com/r/5iujo7

Link:
Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Ma - Benzinga

By David Willey, Benzinga

Melville, NY --News Direct-- BioRestorative Therapies, Inc.

Biotechnology companies have broken many medical barriers in the past 40 years, harnessing the technological revolution to bring innovative solutions to medical problems.

One flourishing field in biotech is regenerative medicine, a market worth $16.9 billion in 2021. Regenerative medicine harnesses the bodys amazing ability to heal itself, using cutting-edge technology to apply this regenerative power to prompt the body to recover from diseases previously uncured.a

An estimated one-third of Americans would benefit from regenerative therapeutic cures. There are wide applications for such regenerative therapy, with categories including stem cell research, gene therapy and tissue engineering. Some difficulties for regenerative medicine companies include arduous Food and Drug Administration (FDA) trial processes and the need for vertical integration of their product development to cut expenses.

Biotech companies involved in regenerative medicine include Mesoblast Ltd. (NASDAQ: MESO), Brainstorm Cell Therapeutics Inc. (NASDAQ: BCLI), Lineage Cell Therapeutics (NYSE: LCTX) and BioRestorative Therapies Inc. (NASDAQ: BRTX).

Here is a look at some companies looking to be leaders in the regenerative therapy field:

Mesoblast Ltd. develops novel treatments for back pain and various cardiovascular conditions. This Australia-based company focuses primarily on cell therapy solutions, with a mesenchymal lineage stem cell (MSC) technology platform. This develops MSCs, highly multipotent cells taken from healthy bone marrow, and develops treatments for tissue damage, heart disease and more.

Lineage Cell Therapeutics is a company pioneering cell-based therapies to treat serious diseases, including ocular disorders and cancer. It uses its proprietary cell-therapy platform to develop and manufacture self-renewing stem cells into differentiated cells, which can be transplanted to treat problems including cancer or degenerative diseases.

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Brainstorm Therapeutics focuses on cell therapies for neurodegenerative diseases. Its autologous cellular therapeutics platform NurOwn treats the disease by differentiating the patients healthy MSCs. Brainstorms work may eventually provide treatments for such neurodegenerative diseases as Alzheimers.

BioRestorative Therapies, which primarily develops products using highly therapeutic adult stem cells, focuses on disc/spine disease and metabolic disorders. BioRestoratives brtxDisc program is developing a treatment for the millions of Americans suffering from either chronic or acute back pain. Its product BRTX-100, which uses autologous stem cells to treat degenerative spinal discs, is in a Phase Two FDA trial.

BioRestorative is also tackling obesity, which currently affects over 40% of Americans. It is developing the product ThermoStem, which harnesses the bodys natural production of healthy brown fat cells to target patient obesity and other metabolic issues associated with obesity.

BioRestorative believes that its treatments will also help condition the body for better future regeneration and responses to medical treatment. A significant advantage for the company is the vertical integration of development and production it has through its clinical-grade cell therapy manufacturing facility. This facility, completed in April, gives BioRestorative control and oversight in the cell manufacturing process, apart from the flexibility to make its own decisions and to correct quality issues in real-time. Owning the facility mitigates the expense normally associated with these activities, which is a great benefit when conducting FDA trials.

Learn more about BioRestorative by visiting its website.

BioRestorative Therapies was founded by scientists and researchers committed to developing stem cell therapies to address unmet needs in patients with highly prevalent conditions.Our advances in stem cell biology and delivery protocols harbor great promise in conditioning our bodies own regenerative potential to treat major diseases more effectively than current interventions.Today, BioRestorative is actively developing programs that aim to dramatically increase quality of care for both (i) chronic back pain caused by disc degeneration, as well as (ii) metabolic disorders including obesity and diabetes.

This post contains sponsored advertising content. This content is for informational purposes only and is not intended to be investing advice.

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A Look At Some Of The Companies Innovating In the Cutting-Edge Regenerative Medicine Field - Yahoo Finance

As the 3rd presenter during the morning session of the American Society for Dermatologic Surgery Meeting, Emerging Concepts, Saranya Wyles, MD, PhD, assistant professor of dermatology, pharmacology, and regenerative medicine in the department of dermatology at the Mayo Clinic in Rochester, Minnesota, explored the hallmarks of skin aging, the root cause of aging and why it occurs, and regenerative medicine. Wyles first began with an explanation of how health care is evolving. In 21st-century health care, there has been a shift in how medical professionals think about medicine. Traditionally,the first approach was to fight diseases, such as cancer, inflammatory conditions, or autoimmune disorders. Now, the thought process is changing to a root cause approach with a curative option and how to rebuild health. Considering how to overcome the sequence of the different medications and treatments given to patients is rooted in regenerative medicine principles.

For skin aging, there is a molecular clock that bodies follow. Within the clock are periods of genomic instability, telomere attrition, and epigenetic alterations, and Wyles lab focuses on cellular senescence.

We've heard a lot atthis conference about bio stimulators, aesthetics, and how we can stimulate our internal mechanisms of regeneration. Now, the opposite force of regeneration isthe inhibitory aging hallmarks which include cellular senescence. So, what is cell senescence? This isa state that the cell goes into, similar to apoptosis or proliferation, where the cell goesinto a cell cycle arrest so instead of dividing apoptosis, leading to cell death,the cell stays in this zombie state, said Wyles.

Senescence occurs when bodies require a mutation for cancers. When the body recognizes there is something wrong, it launches itself into the senescent state, which can be beneficial. Alternatively, chronic senescence seen with inflammageing, like different intrinsic markers, extrinsic markers, and UV damage, is a sign of late senescence. Senescence cells can be melanocytes, fibroblasts, and cells that contribute to the regeneration of the skin.

I think were in a very exciting time ofinnovation and advancements in medicine, which is the meeting of longevity science of aging and regenerative medicine, said Wyles.

Regenerative medicine is a new field of medicine that uses native and bioengineered cells, devices, and engineering platforms with the goal of healing tissues and organs byrestoring form and function through innate mechanisms of healing.Stem cell therapy and stem cell application are commonly referenced with regenerative medicine. Typically, first-in-class treatments include cells, autologous or allogeneic, different types of cells that areassociated with high-cost due to the manufacturing.

With regenerative medicine, there's a new class of manufacturing. Regenerative medicine is not like traditional drugs where every product is consistent. These are cells, so the idea of manufacturing, and minimally manipulating, all comes into play. Now, there's a new shift towards next-generation care. This is cell-free technology. So, this is the idea of exosomes, because these are now products from cells that can be directly applied, they can be shelf-stable, accessible, and more cost-effective, said Wyles.

Exosomes are the ways that the cells communicate with each other. Cells have intercellularcommunications and depending on the source of the exosomes, there can be different signals. Wyles focused specifically on a platelet product, which is a pooled platelet product that can be purified and used for different mechanisms including wound healing, fat grafting, degenerative joint disease, and more.In a cosmetic studyconducted by Mayo Clinic, a topical platelet exosome product was applied to the face in the morning and the evening. Application included a 3-step regimen, a gentle cleanser, a platelet exosomeproduct, and then a sunscreen.

After 6 weeks, there was a significant improvement in redness and a 92% improvement in the hemoglobin process. Vasculature also improved across age groups. The study enrolled 56patients, and the average age was 54. Patients in their 40s, 50s, and 60s saw consistent improvement in redness and skin aging.

Lastly, Wyles stressed that as dermatologists think through the science-driven practices of these innovative strategies for skin aging, wound healing, and other regenerative approaches, they must think about responsible conducts of research. Currently, there are no FDA indications for exosomes being injected.

Reference:

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The Switch to Regenerative Medicine - Dermatology Times

Carlsbad, CA, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The Alliance for Regenerative Medicine (ARM), the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies, today announced the election of its 2023 Officers, Executive Committee, and Board of Directors.

The announcement comes as ARM kicks off its 2022 Cell & Gene Meeting on the Mesa, a gathering of 1,800 leaders in the cell and gene therapy sector.

The Executive Committee and Board of Directors oversee the formation and execution of ARMs strategic priorities and focus areas. These distinguished leaders are instrumental to ARMs leadership of the sector.

We are delighted to welcome our 2023 Officers, Executive Committee members and Board of Directors, said ARMs Chief Executive Officer Timothy D. Hunt. The pipeline of transformative cell and gene therapies will continue to accelerate in 2023, creating more urgency to ensure that patients have access to life-changing medicines. ARMs Board of Directors and our more than 450 member organizations globally are vital to this mission.

ARM 2023 Officers:

Devyn Smith, Ph.D. Chief Executive Officer, Arbor Biotechnologies (Chair)

Dave Lennon, Ph.D. Chief Executive Officer, Satellite Bio (Vice Chair)

Alison Moore, Ph.D. Chief Technology Officer, Allogene Therapeutics (Secretary)

Chris Vann Senior Vice President, Chief Operations Officer, Autolus (Treasurer)

ARM 2023 Executive Committee:

Devyn Smith, Ph.D. Chief Executive Officer, Arbor Biotechnologies (Chair)

Dave Lennon, Ph.D. Chief Executive Officer, Satellite Bio (Vice Chair)

Alison Moore, Ph.D. Chief Technology Officer, Allogene Therapeutics (Secretary)

Chris Vann Senior Vice President, Chief Operations Officer, Autolus (Treasurer)

Bob Smith, MBA Senior Vice President, Global Gene Therapy Business, Pfizer

Miguel Forte, M.D., Ph.D. Chief Executive Officer, Bone Therapeutics

Laura Sepp-Lorenzino, Ph.D. Executive Vice President and Chief Science Officer, Intellia Therapeutics

Arthur Tzianabos, Ph.D. Chair of the Board, Homology Medicines

ARM 2023 Board of Directors

* New to the Board for 2023

* Faraz Ali, MBA Chief Executive Officer, Tenaya Therapeutics

Robert Ang, MBBS, MBA Chief Executive Officer, Vor Biopharma

* Catherine Bollard, M.B.Ch.B., M.D. Director of the Center for Cancer and Immunology Research, Childrens National Hospital and The George Washington University

Amy Butler, Ph.D. President, Biosciences, Thermo Fisher

Bradley Campbell, MBA President and Chief Executive Officer, Amicus Tx

Miguel Forte, M.D., Ph.D. Chief Executive Officer, Bone Therapeutics

* Christine Fox President, Novartis Gene Therapies

Bobby Gaspar, M.D., PhD. Chief Executive Officer, Orchard Therapeutics

Jerry Keybl, Ph.D. Senior Director, Cell & Gene Therapy, MilliporeSigma

Brett Kopelan Executive Director, Debra of America

* Ann Lee, Ph.D. Chief Technical Officer, Prime Medicine

Dave Lennon, Ph.D. Chief Executive Officer, Satellite Bio

Tim Lu, M.D., Ph.D. Chief Executive Officer and Co-Founder, Senti Biosciences

John Maslowski, M.S. Chief Commercial Officer, Forge Biologics

Chris Mason, M.D., Ph.D. Founder & Director, Ori Biotech

Debra Miller Founder & Chief Executive Officer, CureDuchenne

Alison Moore, Ph.D. Chief Technology Officer, Allogene

Adora Ndu, PharmD, J.D. Chief Regulatory Officer, BridgeBio

Susan Nichols President & Chief Executive Officer, Propel BioSciences

Emile Nuwaysir, Ph.D. Chief Executive Officer, Ensoma

Karah Parschauer, J.D. Chief Legal Officer, Ultragenyx

* Jacob Petersen Corporate Vice President and Head of Stem Cell Research & Development, Novo Nordisk

Louise Rodino-Klapac, Ph.D. Executive Vice President, Head of Research & Development, Chief Scientific Officer, Sarepta Therapeutics

Jeff Ross, Ph.D. Chief Executive Officer, Miromatrix Medical

* Alberto Santagostino Senior Vice President, Head of Cell & Gene Technologies, Lonza

Laura Sepp-Lorenzino, Ph.D. Executive Vice President & Chief Scientific Officer, Intellia Therapeutics

R.A. Session, MBA, MSF President, Founder & Chief Executive Officer, Taysha Tx

Curran Simpson, M.S. Chief Operations and Chief Technical Officer, REGENXBIO

Bob Smith, MBA Senior Vice President, Global Gene Therapy, Pfizer

Devyn Smith, Ph.D. Chief Executive Officer, Arbor Biotechnologies

Arthur Tzianabos, Ph.D. Chair of the Board, Homology Medicines

Christopher Vann Senior Vice President & Chief Operating Officer, Autolus Therapeutics

Kristin Yarema, Ph.D. Chief Commercial Officer, Atara Bio

Continued here:
The Alliance for Regenerative Medicine Announces Election of 2023 Officers, Executive Committee, and Board of Directors - GlobeNewswire

Until recently, researchers could not see gene expression in an individual cell. Thanks to single cell sequencing techniques, they now can. But the timing of changes is still hard to visualize, as measuring the cell destroys it.

To address this, we developed an approach based on models in basic physics, explained Welch, treating the cells like they are masses moving through space and we are trying to estimate their velocity.

The model, dubbed MultiVelo, predicts the direction and speed of the molecular changes the cells are undergoing.

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Our model can tell us which things are changing firstepigenome or gene expression--and how long it takes for the first to ramp up the second, said Welch.

They were able to verify the method using four types of stem cells from the brain, blood and skin, and identified two ways in which the epigenome and transcriptome can be out of sync. The technique provides an additional, and critical, layer of insight to so called cellular atlases, which are being developed using single cell sequencing to visualize the various cell types and gene expression in different body systems.

By understanding the timing, Welch noted, researchers are closer to steering the development of stem cells for use as therapeutics.

One of the big problems in the field is the artificially differentiated cells created in the lab never quite make it to full replicas of their real-life counterparts, said Welch. I think the biggest potential for this model is better understanding what are the epigenetic barriers to fully converting the cells into whatever target you want them to be.

Additional authors on this paper include Chen Li, Maria C. Virgilio, and Kathleen L. Collins.

Paper cited: Single-cell multi-omic velocity infers dynamic and decoupled gene regulation, Nature Biotechnology. DOI: 10.1038/s41587-022-01476-y

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Mathematical model could bring us closer to effective stem cell therapies - Michigan Medicine

As a top regulator at the Food and Drug Administration, Peter Marks isnt responsible for weighing the cost of the treatments his teams review. But he is worried that some of the drug industrys most promising medicines may not reach patients with uncommon diseases if companies cant figure out how to sell them.

There are an estimated 7,000 rare diseases, many of which affect only small groups of people. Genetic medicines, including RNA-based drugs and gene replacement therapies, could offer a powerful way to treat, and potentially even cure, some of them. But for would-be developers, diseases affecting only a few dozen people might not represent a large enough market to justify the cost of developing and selling a new treatment.

We're not going to find enough philanthropic groups to foot the bill for gene therapies for the hundreds upon hundreds of different diseases that need to be addressed, said Marks, head of the FDAs Center for Biologics Evaluation and Research, at a conference hosted by the Alliance for Regenerative Medicine on Wednesday.

We're gonna have to find a way to make this commercially viable so that industry can find a way forward towards this."

According to Marks, commercial viability for a gene therapy means administering roughly 100 to 200 treatments a year, a threshold that could be difficult to clear in a single country for rare conditions like severe combined immunodeficiences or adrenoleukodystrophies.

It has not escaped our attention at FDA that there have been some clouds on the horizon in gene therapy, said Marks, noting instances when gene therapies were taken off the market or returned by their developers to the original academic researchers.

In Europe, for example, first GSK and then Orchard Therapeutics abandoned one of the first gene therapies approved there, a treatment called Strimvelis for a condition known as ADA-SCID. Only a few dozen patients were ever treated, and Orchard has also handed back rights to a successor treatment. More recently, Bluebird bio withdrew two gene therapiesfrom the EU market after running into difficulties securing reimbursement in several European countries.

Bluebird recently won FDA approval for both of those therapies in the U.S. One, to be sold as Skysona at a cost of $3 million, is for an inherited condition known as CALD that affects about 50 boys each year. Bluebird has said it expects to treat around 10 each year.

In his remarks to the conference, known as the Meeting on the Mesa and attended by many in the cell and gene therapy field, Marks highlighted a few areas where the FDA could help ease hurdles for ultra-rare disease treatments.

The agency is currently putting together a cookbook for developing and manufacturing of bespoke gene therapies, which could help academic groups more easily transfer treatments theyre working on to industry. Its also looking into how to use non-clinical and manufacturing data from one application to speed the review of others that share similar technology.

There are certain pieces of gene therapies that are not like your typical small molecule drug, because they're reused repeatedly, Marks said.

Automated manufacturing could be another solution to help lower the costs of production, which are significantly higher for cell and gene therapies than for other more established drug types.

The FDA is also hoping to get on the same page with other regulators so that developers could be more confident a product they gain approval for in one country would have a good chance of success in others.

Some of [these problems] may relate to how we can make gene therapies for small populations more widely available, Marks said. What may be a tiny population in the U.S. becomes a reasonable sized population when you go globally.

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'We have to find a way': FDA seeks solutions to aid bespoke gene therapy - BioPharma Dive

Free virtual conference for World Cord Blood Day 2022 (November 15th) to feature renowned transplant doctors and researchers presenting ground-breaking work in cellular therapy using cord blood to treat cancer, HIV, cerebral palsy and autism. In addition, bestselling author, Delia Ephron, to present her memoir about fighting leukemia, "Left on 10th: A Second Chance at Life."

TUCSON, Ariz., Oct. 13, 2022 /PRNewswire/ -- Innovations using cord blood in cellular therapy, including traditional stem cell transplants and regenerative medicine research, will be featured during World Cord Blood Day (WCBD) 2022. Recommended for healthcare professionals, expectant parents and STEM students, the official virtual conference, hosted by Save the Cord Foundation, will be held on November 15th (register free via Eventbrite).

We are proud to announce the following speakers:

View the full agenda here: https://www.worldcordbloodday.org/online-medical-conference-agenda-wcbd-2022.html

Hosted and organized by Save the Cord Foundation, a 501c3 non-profit, World Cord Blood Day 2022 brings together the cord blood community through numerous live events and activities held around the globe and online. QuickSTAT Global Life Science Logistics, recognized leader in medical shipping and healthcare logistics, is the Official Sponsor. Inspiring Partners include Be the Match (NMDP), World Marrow Donor Association (WMDA-Netcord), Association for the Advancement of Blood and Biotherapies (AABB), Cord Blood Association (CBA), and Foundation for the Accreditation of Cellular Therapy (FACT).

Visit http://www.WorldCordBloodDay.org to register free for the online conference and learn how you can participate on-line or at an event locally in your community (#WCBD22 #WorldCordBloodDay).

About Save the Cord Foundation (Organizer and host of WCBD 2022)

Save the Cord Foundation (a 501c3 non-profit) was established to advance cord blood education. The Foundation provides non-commercial information to parents, health professionals and the public regarding methods for saving cord blood, as well as current applications using cord blood and the latest research. Learn more at http://www.SaveTheCordFoundation.org.

About QuickSTAT Global Life Science Logistics (Official Sponsor of WCBD 2022)

Every day, QuickSTAT, a part of Kuehne+Nagel, safely and reliably moves thousands of critical shipments around the world. For over forty years, QuickSTAT has been entrusted with transporting human organs and tissue for transplant or research, blood, blood products, cord blood, bone marrow, medical devices, and personalized medicine, 24/7/365. QuickSTAT's specially trained experts work with hospitals, laboratories, blood banks and medical processing centers, and utilize the safest routes to ensure integrity, temperature control and chain of custody throughout the transportation process. Learn more at http://www.quickstat.aero.

Media Contact:

Charis Ober

(520) 419-0269

[emailprotected]

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Discover Medical Advances in Cellular Therapy Research Using Cord Blood for Cancer, HIV, Cerebral Palsy and Autism During World Cord Blood Day 2022 -...

MIAMI, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The American Academy of Stem Cell Physicians will be hosting its fall Scientific Congress in Chicago, IL, on Oct. 28-30, 2022. The conference will feature three days of educational and networking events with leading physicians from across the fields of stem cells, live cells, and regenerative medicine. A Board Examination process will be available, creating a pathway for participants to earn a Diplomat and Fellowship Certification in Regenerative Medicine.

The Board of American Academy of Stem Cell Physicians is the official board certifying body of the American Academy of Stem Cell Physicians(AASCP). As a nationally recognized academy with a mission to bring like-minded physicians together to increase awareness and education for the evolving field of regenerative medicine, the AASCP is proud to announce its Fellowship and Diplomat Certification.

In order to be eligible for certification or recertification through the AASCP, licensed physicians in good standing must meet the stringent eligibility requirements that have been defined by the board. AASCP places an emphasis on not only psychometrically evaluated testing and advanced training, but also moral character and experience. Furthermore, AASCP has a clear path toward recertification for qualified physicians. Their standards for recertification include a commitment to continuing medical education, successful completion of a recertification examination, participation in a non-remedial medical ethics program, and additional requirements.

AASCP is known for working with physicians to provide unique opportunities for board certification in their specialty of regenerative medicine. Specifically, the AASCP offers ongoing workshop modules led by esteemed physicians in this field who certify and educate on different treatment approaches and techniques. Another defining characteristic of the AASCP is theircommitment to ongoing education and awareness. To support this goal, the AASCP has developed innovative committees, including its Institutional Review Board and created opportunities for physicians and researchers to submit their work for peer review and exposure.

The AASCP was founded to recognize licensed physicians who have shown a specialty and interest in regenerative medicine. Increasingly, hospitals and medical staff placement agencies are prioritizing hiring Board-Certified Physicians. For this reason, the AASCPfeels it is important to offer qualified professionals a choice when they're researching board certifying bodies.

The American Academy of Stem Cell Physicians (AASCP) is an organization created to advance research and the development of therapeutics in regenerative medicine, including diagnosis, treatmentand prevention of disease related to or occurring within the human body. Secondarily, the AASCP aims to serve as an educational resource for physicians, scientistsand the public in diseases that can be caused by physiological dysfunction that areameliorableto medical treatment.

For further information, please contact WilsonDemenessez at 305-891-4686, and you can also visit us at http://www.aascp.net.

Contact Information: Wislon Demenessezz AASCP account Sales manager wilson@genorthix.com 305-891-4686

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American Academy of Stem Cell Physicians to Offer Licensed Physicians Board Examination in Regenerative Medicine - GlobeNewswire

As activity in this space grows, so do the hurdles in moving these products forward.

Cell and gene therapy (CGT)its risks and promisesare succinctly summarized in this description of clinical trial number NCT01129544, a Phase I/II study in children born with X-linked severe combined immunodeficiency (SCID-X1), an inherited, rare, and life-threatening disease. The eight-person trial, which began in May 2010, continues today. The following paragraph has been edited.1

Gene transfer is still research for two reasons. One, not enough children have been studied to tell if the procedure is consistently successful. [And] we are still learning about its side effects and doing gene transfer safely. In previous trials, five children developed gene transfer-related leukemia; four are in remission; one died.

If the above information has stifled the research communitys scientific curiosity about CGT, it is not evident. Evidence from numerous sourcesClinicalTrials.gov, the Alliance for Regenerative Medicine (ARM), FDAare chock-a-block with studies, trials, and figures showing these therapies popularity. In the second quarter of 2022, 3,633 such treatments were in development, up from 1,745 in May 2021. The vast majority are in the preclinical stage.2,3

Some sources are revealing more.

Most indicate that academics now have a remarkable presence in the CGT development space, including sponsorship. Last year, for the first time, ARM included sponsorship figures in its twice-annual industry report.4 Academic- and government-sponsored trials far exceeded industry for sponsored trials in CGT. Stephen Majors, senior director for public affairs, ARM, says the alliance knew of academias presence for the past few years, but only was able to get data this year from its partner, Global Data.

Less reliable, but still noteworthy, are data from ClinicalTrials.gov: for active Phase I trials, industry has 89; others, which covers academia and government, have 50. Industry enrollment for Phase I is 172; others, 116.Phase III is one for others, eight for industry.

A little disruption in pharmas corner of the world? It seems that way. While basic bench to preclinical to clinical trial has long been the traditional route to FDA approvaland no one interviewed for this article suggested a reroutewhat it does imply is that pharma members have some competition from the spin-offs and academic biotechs that historically they have absorbed.

There are suspected trends that we are watching, says Majors.As to whether academias presence in this spot can be called a trend depends on ones definition of what a trend is. The Centers for Disease Control and Prevention (CDC) considers changes over a number years to determine a trend; financial investment firms typically evaluate over a two-year period.Considering that CGT companies raised $23.1 billion in 2021, 16% more than 2020,3 the answer to the above question could be, maybe.

The CGT space is still immature, according to Mike Rea, founder of Protodigm, a self-described exploratory research organization that partners with biopharma clients on alternative development and commercial solutions. Physicians need time to be comfortable with these therapies, notes Rea, so they may not be used on a regular basis.

For example, physicians have to understand how to deliver the gene, agrees cardiologist Arthur M. Feldman, MD, PhD, whose lab worked on a heart failure-related mutation in BAG3 for decades.

Last month, the company he founded, Renovacor, agreed to be acquired by Rocket Pharmaceuticals.5 We are asking physicians to do something they never did before and to understand a very different set of information, including risk/benefit discussions that they didnt learn about in medical school, he says. Feldman is a Laura H. Carnell Professor of Medicine, Division of Cardiology, and a member of the Center for Neurovirology and Gene Editing at the Lewis Katz School of Medicine at Temple University.

Chris Learn, Parexels vice president of cell and gene therapy, is unequivocal regarding academias increased presence in the drug development space focused around these treatments. He cites MD Anderson and Moffitt Cancer Center as two institutions that are sponsoring their own trials. The lines are really blurring here, he tells Applied Clinical Trials. It is indisputable.

The following is a look at how academia is showing up in various reports.

In its 2022 report4, ARM separated sponsorship, type of therapygene, cell-based, and tissue engineeringand trial phase. What these data show are industry far exceeding academic and government sponsored trials for gene therapy, while for cell therapy alone, the reverse is true: 656 cell therapy trials for academic and government, and 424 for industry. For gene therapy, there are 84 for the academics and government, and 222 for industry. In a later report, ARM found non-industry trials dropped.

Pharma Intelligences Pharma R&D Annual Review does not break down trials by their sponsors. It does, however, break down whats in the pipeline in various categories, including by the number of therapies per company, and by disease type.6 In numbers captured prior to March 2020, the analysis reported 1,849 companies with asingle drug in its pipeline, up from 1,633 in 2019, comprising more than half of all drug companies. As for types of therapies, gene therapy was in third place, the same spot it occupied in 2019. (Cancer-related therapies occupy the top spots.) Overall, biotech therapies in the pipeline increased by 13.2% in 2020 over 20196,135 vs. 5,422. Cellular therapy, the field in which academia is dominating, rose to 14th place, up from 33.

In 1982, Feldman was a resident in the cardiac care unit at the Johns Hopkins Hospital in Baltimore when he took care of a 22-year-old woman, a native Pennsylvanian, who was dying of heart failure. Sadly, we didnt have drugs with which to treat her, he recalls. Feldmans involvement with the case and the womans family led to his career as a cardiologist, he says. Twenty years later in Philadelphia, he was asked to see a heart-failure patient in consult, who turned out to be the aunt of the younger woman. It would take almost another 10 years until the technology became available to identify the genomic anomaly in this family. Here, a genetic variant that is produced by one of two alleles causes the protein product to be unstable. The result: the cell removes it, so the person with the variant has just half the amount of required protein.

BAG3 is an interesting protein that is found in the heart, the skeletal muscles, and the nervous system, including the brain. Its function is to help remove degraded and misfolded proteins, stop apoptosis or programmed cell death, and maintain the structure of the skeletal muscles. A missing allele isnt the only genetic cause for heart failure, Feldman said. Other patients, while having the correct amount of DNA, have a point mutationa single amino acidin half of the produced DNA. That single letter is the wrong amino acid in the specific site in the protein.

Around this time, Kamel Khalili, PhD, Laura H. Carnell Professor, and chair of the department of microbiology, immunology, and inflammation; director of the Center for Neurovirology and Gene Editing; and director of the Comprehensive NeuroAIDS Center, Lewis Katz School of Medicine, Temple University, had created a method by which he could excise the HIV virus from patients using the new technique of CRISPR-Cas9.

Khalili believes that BAG3 may be involved in the pathogenesis of HIV-1 in brain diseases and protein quality control caused by viral infection as well as several other disorders, including Alzheimers disease and dementia. BAG3 changes the homeostasis of the cell, he says. The only solution is to fix the cell. Khalili has used CRISPR technology to excise the viral genome in both small and large investigational animals and has recently started a Phase I trial to test the safety of the new gene-editing treatment. Khalili, too, started a company, but Temple holds the license. In the case of Renovacor, it was granted the license by Temple.

As a scientist, when you are doing something in biomed research, [the] goal is to translate bench work to the clinic for [the] wellness of people. We are doing long hours and long days because we want to help. We are trying to see if discovery can help people, says Khalili. I know my limit, I stop at business aspects. My interest is to discover research which can help populations.

Was Feldman happy with his business experience? As a company gets bigger, others join the team who fulfill other roles, like acquiring funding or developing the actual product, he says. Releasing the control reins are difficult. But if it speeds up the timeline to get an approved product into the clinic, then its all worth it, he adds.

Researchers such as Feldman and Khalili, says Kaspar Mossman, PhD, director of communications and marketing at QB3, a University of California biotech accelerator, are normally not deeply interested in business. He notes the new flagship space in UC Berkeley called Bakar Lab. So far, it has 25 companies, one-third from university labs. They collaborate, they share equipment, [at times] they merge, Mossman tells Applied Clinical Trials.

And, he adds, Academics tend to be very smart individuals. The more time they spend in business, they learn stuff and become serial founders, says Mossman. They are honest about not wanting to be a CEO.

In terms of business, the academics employers are also pretty smart. The huge bugaboo with CGT commercialization is the manufacturing processthe need for an apheresis unit, ultra-cold storage, and regulated cell processing facilities.

Some institutions are building their own manufacturing facilities to more easily meet the increasingly complicated standards pertaining to regenerative medicine production. Harvard, MD Anderson, Moffitt, the University of Pennsylvania, and the University Hospital of Liege in Belgium8 all have or are planning to build their own facilities.

As for how academias presence impacts the traditional pharma space, those interviewed cited pros and cons. More research is better, more companies vying for venture capital funding is not. But more trials mean more competition among similar therapies, which, says Majors, is a good thing.

We need experimentation, adds Rea. If left to pharma, he says, the research wouldnt happen. Smaller biotechs are taking the risk. Over the last 10 years, Rea believes pharma has been slow in the risk-taking department. Once upon a time, pharma didnt have many competitors. Now, with many numerous smaller companies with viable assets, willing to accept a smaller net profit, the competition is creating some angst. Pharma cant project everyones movement, says Rea. The gene/cell therapy landscape [for products] is huge.

Likely adding to the angst: Those smaller biotechs are getting financial help. Between April 4, 2021, and June 24, 2021, of 23 start-up financing deals, 19 involved academics.2

Learns viewpoint is different. He says there are too many players out there, and while large pharma may be averse to risk, I really do believe what we are witnessing are simply market forces that have played into this. There is so much cash coming in, he continues, that people can be blinded by the pitfalls. The CGT area, he adds, is bloated and he says the industry needs an overall strategy.

Learn doesnt think that academias presence in the CGT space is a flash in the proverbial pan. The enthusiasm to find cures is real, and some research institutions have the endowments to see the trials through. I think it is just the beginning, says Learn. Academia will put their futures in front of them. Why put all your sweat equity into it and not have any fiduciary benefit of the approved product?

In Pharma Intelligences 2020 Pharma R&D Review, its author questioned the wisdom of so many drugs, overall, in the pipeline4,001 added in 2018 and 4,730 added in 2019, for a total of 17,737 drug candidates. [A]re the industrys eyes getting too big for its belly? Unless it can continue to provide [approved therapies] then a certain degree of control in the pipeline might be advisable, the report stated.6

And now to costs. While no one doubts these cures change lives, the question of access persists. FDAs approval of Bluebird Bios second therapy this year, branded as Skysona, for early but active cerebral adrenoleukodystrophy, is expected to cost $3 million. Learn doubts that payers are jumping up and down to get Skysona on their formularies.

Its still a fairly dicey business proposition for companies to invest in this field, Steven Pearson, MD, president of the Institute for Clinical and Economic Review (ICER), said recently.8Theres still a risk that next-generation therapies will not flourish even in developed countries health systems, he added.

One positive development in the US, however, occurred late last month when Congress reauthorized the Prescription Drug User Fee Act (PDUFA) for the next five years, 2023-2027. The action maintained FDAs authority to collect fees from manufacturers and keep and recruit agency staff to review the increased number of CGT applications. Majors says most of FDAs review of CGT products involves scalability and consistent reproducibility in the manufacturing process, which, of course, means traveling.

According to a Senate press release9, FDA is seeking to hire at least 320 new staff members. In a statement, Pharmaceutical Research and Manufacturers of America (PhRMA) said a modern regulatory framework supported by PDUFA helps ensure patients have timely access to lifesaving medicines.

PDUFA reauthorization aside, there is little argument that the field of CGT, from research and drug discovery through commercialization, is advancing rapidly. In turn, so are the unique operational and manufacturing challenges that these therapies present. This reality may thin the currently crowded playing field in CGT going forward, with those sponsors and partners best prepared to deliver on the numerous touchpoints required separating from the pack.

Christine Bahls, Freelance Writer for Medical, Clinical Trials, and Pharma Information

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The Risk-Reward Proposition for CGT Clinical Trials - Applied Clinical Trials Online

This week: Vita Therapeutics raises US$31 million in Series B funding to progress cell therapies for neuromuscular diseases to clinic and expand its discovery pipeline, Cellusion and Minaris enter business alliance to manufacture cell therapy for bullous keratopathy and Ray Therapeutics and Forge Biologics expand partnership to include plasmid DNA manufacturing.

Vita Therapeutics (MD, USA) announced it has secured US$31 million in Series B financing to accelerate its lead program VTA-100, for limb-girdle muscular dystrophy, to the clinic. The funding will also be utilized to develop Vitas newest program VTA-120 to treat facioscapulohumeral muscular dystrophy and to continue expansion of the companys discovery pipeline. The financing was led by Cambrian Biopharma (NY, USA) and Solve FSHD (Vancouver, Canada).

Douglas Falk, CEO at Vita Therapeutics stated: The support from this strategic group of quality investors further validates Vitas cell therapy platform and our mission to bring transformative therapies that target the root cause of disease to patients with muscle disorders and cancers. This syndicates confidence in our ability to further progress our programs is energizing and we are thrilled to have them as partners. We are making notable progress with our investigational IND-enabling studies for VTA-100 and are on track to reach the clinic with this important therapeutic candidate within 18 months. Additionally, we are excited to further expand our pipeline to include VTA-120 for the treatment of patients with FSHD. Im incredibly proud of our entire team and the steady momentum we continue to have.

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Cellusion Inc. (Tokyo, Japan) and Minaris Regenerative Medicine (NY, USA) announced a letter of intent for the manufacturing of CLS001, Cellusions novel cell therapy treatment for bullous keratopathy. CLS001 utilizes corneal endothelial cell substitute from induced pluripotent stem cells for regeneration of the corneal endothelium. Under the letter of intent, the two companies will together develop the manufacturing process of CLS001 and Minaris will optimize processes to meet FDA requirements.

Shin Hatou, CEO of Cellusion stated: We are very enthusiastic to have the partnership with Minaris, a well-established regenerative medicine partner with over 20 years experiences including the predecessor companies, Progenitor Cell Therapy and Hitachi Chemical, and one of the leading CDMOs in the U.S. since the dawn of the field. Together, we make our best efforts to develop the robust manufacturing process of CLS001 for patients suffering from bullous keratopathy due to the cornea donor shortage all over the world.

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Ray Therapeutics (CA, USA) and Forge Biologics (OH, USA) announced their existing manufacturing partnership will expand to include clinical stage plasmid DNA production to support Ray Therapeutics retinitis pigmentosa gene therapy program, RTx-015. The program currently utilizes Forges platform of manufacturing processes, including its proprietary HEK 293 suspension Ignition Cells and pEMBR adenovirus helper plasmid. To further aid the RTx-015 program, Forge will now also provide plasmid manufacturing services, as well as adeno-associated viral vector process development, scale-up engineering, and cGMP manufacturing services.

Paul Bresge, CEO of Ray Therapeutics stated: By adding clinical grade plasmid production to their existing suite of AAV manufacturing capabilities, Forge is easing the scope of production and accelerating the development of our lead therapeutic. Offering everything we need under one roof integrates our entire process so that we can focus on our mission to restore vision in patients losing their sight as fast as possible.

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Cell therapy weekly: Ray Therapeutics and Forge Biologics expand partnership - RegMedNet

CBER maps modernization plan to handle surge in research and applications.

FDAs Center for Biologics Evaluation and Research (CBER) is updating how it manages a growing volume of cellular and gene therapy development programs, seeking added resources and revisions in its oversight of these cutting-edge therapies. Most visible in the elevation of CBERs Office of Tissues and Advanced Therapies (OTAT) into a new super Office of Therapeutic Products (OTP). The change aims to improve functional alignment, increase review capabilities, and add expertise on new cell and gene therapies by establishing multiple branches and divisions in the expanded regulatory unit, as announced in the Federal Register on Sept. 28, 2002.

Stated goals are to help CBER address the substantial growth in innovative, novel products that present new scientific, medical and regulatory challenges that require changes to its structure, including strategies to advance the Regenerative Medicine Advanced Therapy (RMAT) program. The added resources are needed to oversee more than 2000 development programs involving cellular and gene therapies, many involving innovative testing and manufacturing processes. This soaring workload has over-taxed CBER staffers, resulting in serious difficulties in retaining and hiring capable scientists.

The structural changes at CBER reflect agreed-on plans to hire new staffers with funding from recently reauthorized user fee programs. The PDUFA VII commitment letter calls for an additional 132 new hires for CBER in this coming year and another 48 employees the following year, most to support cell and gene therapy reviews at OTP. The reorganization plan calls for OTP to have seven officesfor therapeutic products, clinical evaluation, review management, pharmacology/toxicology, and two for CMCfor gene therapy and for cellular therapy and human tissues. There will be 14 divisions and 32 branches within those offices, providing attractive supervisory opportunities for both new and experienced staffers.

These changes come in the wake of FDA approval of two new gene therapies that have drawn wide attention for both their therapeutic potential and for million-dollar price tags. Bluebird bios Zynteglo was approved by FDA in August for patients with beta thalassemia, an inherited blood disorder causing serious anemia. That was followed a few weeks later with approval of Bluebirds Skysona to treat a rare neurological disorder afflicting young boys. Zynteglo carries a $2.8 million price tag, Skysonas list price is $4 million, but both therapies are expected to target fewer than 1500 patients, limiting the overall cost impact for the US healthcare system. A greater spending effect would come from FDA approval of a new treatment for sickle cell disease from Vertex Pharmaceuticals and CRISPR Therapeutics, which plan to begin a rolling review by FDA in the coming months. The important potential benefits of these treatments, along with concerns about their impact on healthcare spending and access, speaks to the need for a highly capable and sufficiently resourced FDA oversight program.

These developments also highlight the importance of sound testing and production methods for therapies made from living organisms, which are inherently variable and difficult to control and measure to assure product safety, identify, quality, purity, and strength. The surge in applications from a broad range of firms, moreover, has made it difficult for CBER staffers to schedule formal meetings with each sponsor seeking advice on how best to perform manufacturing and testing processes. And publishing new guidance on these changing and emerging issues also takes time and resources.

In response, FDA looks to engage a broad range of sponsors on topics related to product development through a series of virtual town hall meetings. The first was held Sept. 29, 2022 and addressed how manufacturers should describe and inform FDA about chemistry, manufacturing, and controls (CMC) in applications for gene therapies. Wilson Bryan, OTAT (now OTP) director, opened the session by describing plans for establishing OTP as a super office to increase review capabilities and enhance expertise on gene and cellular therapies and set the stage for OTP branch chiefs to field a broad range of queries, ranging from basic CMC policies for various stages of development, to the scope of potency assays and impact of delivery devices on dose potency and quality [a recording of the town hall meeting is available at the FDA events link].

Main topics were comparability testing, assays for product characterization, and process controls. OTP staffers emphasized the importance of determining process requirements early in development to avoid late changes and analytical method variability that could raise uncertainties likely to delay clinical trials. Products with complex mechanisms of action, they advised, stand to benefit from early product characterization and potency assay development. And developers of gene therapies should use multiple production lots during a clinical study to ensure product consistency and quality, even for treatments for very small patient populations.

Manufacturers raised questions about differing CMC issues between early Phase I and late-stage clinical trials and voiced concerns about product characterization related to autologous cell-based gene therapies. A main theme from FDA was the importance of sponsors establishing a well-controlled manufacturing process and qualified analytical testing well before administering any new gene product. While CBER plans to issue guidance on manufacturing changes and comparability for cellular and gene therapy products, the information provided at this session provides unofficial guidance for implementing changes in product manufacturing and the scope of comparability assessments and development studies expected to support such changes.

Jill Wechsler is Washington editor for Pharmaceutical Technology.

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FDA Expands Oversight of Cell and Gene Therapies - Pharmaceutical Technology Magazine

While sea moss has been harvested and consumed either as food or for healing for thousands of years, there is limited scientific evidence to prove many of these claims, says Carrie Lam, M.D., a physician specializing in family medicine and anti-aging and regenerative medicine at The Lam Clinic in Tustin, California. However, new research into its benefits shows promise in the ability of sea moss to treat inflammation and related disorders, she adds.

There are more studies on the benefits of seaweeds as a whole than on sea moss itself. Preliminary research on sea moss thus far is based on small studies. Large-scale clinical trials are needed to verify the findings listed below.

Your thyroid [a small gland located at the base of your neck] needs iodine to produce thyroid hormones, says Dr. Lam. These hormones, important for proper thyroid function, also play a role in metabolism, she explains. Iodine must be acquired through diet because the body cannot make it on its own.

An iodine deficiency can lead to an enlarged thyroid (or goiter), hypothyroidism and intellectual disabilities in infants and children whose mothers were iodine deficient during pregnancy, according to the American Thyroid Association.

While iodine deficiency has been virtually eliminated in the U.S. since the introduction of iodized salt, even a minor deficiency can cause adverse effects on the thyroid.

In the marine environment, seaweeds are the largest supplier of iodine. However, the iodine content varies among different species, with brown seaweed typically having a greater amount compared to green and red seaweeds. Sea moss in particular contains an average of 3.86 1.49 milligrams of iodine per kilogram dry weight.

Most adults need about 150 micrograms of iodine a day, with pregnant and lactating individuals needing slightly more at 220 micrograms and 290 micrograms, respectively, according to the National Institutes of Health.

An unhealthy gut has an imbalance of gut bacteria. This imbalance or gut dysbiosis is linked to disorders such as inflammatory bowel disease, type 2 diabetes, high blood pressure and cancer.

Prebiotics are a group of fibers that are resistant to digestion. They help stimulate the growth and/or activity of the guts good bacteria.

A review of studies on the effects of seaweeds (including sea moss) as a whole in human, animal and in vitro (microorganisms), suggests that certain components unique to seaweeds have the potential to act as prebiotics and support gut health.The authors of the review note that while these trials show the prebiotic potential of seaweed components, large scale human clinical trials are required to validate findings.

The various nutrients in sea moss not only help build muscles and maintain muscle health but also help maintain a healthy bone structure, notes Dr. Lam. This is of much importance to those on a strict exercise regime or the elderly who tend to see a loss in muscle and bone mass due to the aging process, she says.

A small 2018 observational study looked at the effects of sea moss supplementation in 80 patients with musculoskeletal diseases and joint related symptoms. Subjects were divided into two groups treated with different sea moss supplements.

Some of the study participants from both groups reported beneficial effects such as more strength and energy and less exhaustion and pain after supplement treatment. Researchers concluded that this may be due to the high protein content in sea moss. Notably, subjects given the sea moss supplement with greater protein content had higher muscle energy recovery.

There are other potential health benefits of sea moss. However, as these benefits have only been observed in animal and in vitro studies, further clinical trials on humans are needed.

Tap Into The Health Benefits Of Magnesium

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The Health Benefits Of Sea Moss, According To Experts - Forbes

LEXINGTON, Mass.--(BUSINESS WIRE)--Frequency Therapeutics, Inc. (Nasdaq: FREQ), a clinical-stage regenerative medicine company focused on developing therapeutics to activate a persons innate potential to restore function, today announced that it has completed enrollment of its placebo-controlled Phase 2b study of FX-322 in adults with acquired sensorineural hearing loss (SNHL). The FX-322-208 study, which enrolled 142 individuals, is designed to show improvement in a pre-specified measure of speech perception. The Company plans to release study data in the first quarter of 2023.

I am very pleased with our teams execution of this study for the first potential treatment to restore hearing for those with SNHL. The 208 study was rigorously designed to ensure the stability of an individuals hearing prior to entering the trial and to exclusively enroll those with the types hearing loss where we observed the strongest hearing improvement in prior FX-322 studies. FX-322 continues to have a favorable safety profile and we are aligned with FDA on the primary speech perception endpoint. With a successful outcome of this single-dose study, our intent is to advance the program into Phase 3 trials, said David L. Lucchino, Frequencys chief executive officer.

Mr. Lucchino continued: We are grateful to all the study volunteers, clinicians and site staff for their time and commitment to this trial. We believe the high level of interest from patients and healthcare providers in this study further demonstrates the need for a novel, disease modifying hearing loss treatment to expand the standard of care for the millions of individuals with sensorineural hearing loss.

FX-322-208 Study Design

FX-322-208 is a prospective, randomized, double-blinded, placebo-controlled, multi-center Phase 2b study designed to evaluate the efficacy of a single administration of FX-322 on speech perception in subjects aged 18-65 with hearing loss associated with either noise-induced or permanent idiopathic sudden SNHL. The study enrolled 142 participants, exceeding the original enrollment target of approximately 124, and is being conducted at 28 clinical sites across the US.

The Company previously aligned with the US Food and Drug Administration (FDA) on the use of the specific speech perception primary endpoint. With improved speech perception, individuals may hear words more clearly, a critical unmet need for individuals with hearing loss. The FX-322-208 study is powered at 80% (significance level of 0.05) to observe a statistically significant and clinically meaningful improvement in speech perception at day 90 following dosing, with study responders defined as individuals exceeding the upper 95% confidence interval in the speech perception test. The Company has not publicly disclosed the specific test used for the primary endpoint to maximize the rigor of the study and mitigate potential bias.

During the study, subjects participate in a range of audiologic exams, including pure-tone audiometry, word recognition in quiet, word recognition in noise, the Tinnitus Functional Index (TFI), as well as multiple patient-reported outcome measures including Frequencys proprietary patient reported outcome instrument (RADIAL) in acquired SNHL. All subjects are required to have a documented audiogram from at least six months prior to screening and most patients are evaluated over a 270-day period following dosing. The studys rigorous design includes a lead-in phase with multiple baseline measures. Subjects with instability of baseline tests are disqualified from participation in the study. Study audiometry testing sessions are recorded and monitored by third party audiologists to ensure consistency and identify any anomalies related to how tests were conducted.

In prior studies, the Company observed the greatest concentration of speech perception improvements in individuals with permanent sudden or noise-induced sensorineural hearing loss in the moderate to lower severe hearing loss range. These learnings informed the design and inclusion criteria for the FX-322-208 study. More than 200 individuals have been dosed with a single injection of FX-322 in prior or ongoing studies, and the drug candidate has continued to exhibit a favorable safety profile with no drug-related serious adverse events.

About Sensorineural Hearing Loss

Sensorineural hearing loss is the most common form of hearing loss, typically resulting from damage to sensory hair cells in the cochlea. These cells convert sound waves to signals sent to the brain which are interpreted as speech and sound. Sensory hair cells are lost due to chronic noise exposure, aging, certain viral infections or exposure to drugs that are toxic to the ear. This type of hearing loss impacts around 40 million individuals in the U.S. alone.

About Frequency Therapeutics

Frequency Therapeutics is leading a new category in regenerative medicine that aims to restore human function first in hearing loss and then in multiple sclerosis by developing therapeutics that activate a persons innate regenerative potential within the body through the activation of progenitor cells. Frequencys hearing research focuses on cochlear restoration and auditory repair, and its lead asset, FX-322, is a small-molecule combination product candidate that is the first to show statistically significant and clinically meaningful hearing improvements in clinical trials for sensorineural hearing loss. Frequency is also following early restorative signals in MS to develop medicines with the same underlying regenerative science being brought to hearing loss.

Headquartered in Lexington, Mass., Frequency has an ex-U.S. license and collaboration agreement with Astellas Pharma Inc. for FX-322, as well as additional collaboration and licensing agreements with academic and nonprofit research organizations including Massachusetts Eye and Ear, Mass General Brigham, the Massachusetts Institute of Technology, and the Scripps Research Institute.

For more information, visit http://www.frequencytx.com and follow Frequency on Twitter @Frequencytx.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the timing and design of the Phase 2b study (FX-322-208), including the timing of results and the ability of design features to reduce bias, the commencement of any future FX-322 trials, the interpretation and implications of the results and learnings of other FX-322 clinical studies, the treatment potential of FX-322, estimates of the size of the hearing loss population, the acceptance by the FDA of particular endpoints in the Companys trials, and the potential application of the progenitor cell activation (PCA) platform to other diseases.

These forward-looking statements are based on managements current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of COVID-19 on the Companys ongoing and planned clinical trials, research and development and manufacturing activities, the Companys business and financial markets; the Company has incurred and will continue to incur significant losses and is not and may never be profitable; the Companys need for additional funding to complete development and commercialization of any product candidate; the Companys dependence on the development of FX-322; the unproven approach of the PCA platform and the inability to identify additional potential product candidates; the lengthy, expensive and uncertain process of clinical drug development and regulatory approval; the Companys limited experience successfully obtaining marketing approval for and commercializing product candidates; the results of earlier clinical trials not being indicative of the results from later clinical trials; differences between preliminary or interim data and final data; adverse events or undesirable side effects; disruptions at the FDA and other regulatory agencies; failure to identify additional product candidates; new or changed legislation; failure to maintain Fast Track designation for FX-322 and such designation failing to result in faster development or regulatory review or approval; ability to seek and receive Breakthrough Therapy designation for FX-322; the Companys ability to enroll and retain patients in clinical trials; costly and damaging litigation, including related to product liability or intellectual property or brought by stockholders; dependence on Astellas Pharma Inc. for the development and commercialization of FX-322 outside of the United States; misconduct by employees or independent contractors; reliance on third parties, including to conduct clinical trials and manufacture product candidates; compliance with changing laws and regulations, including healthcare and environmental, health, data privacy and safety laws and regulations; failure to obtain, maintain and enforce protection of patents and other intellectual property rights covering product candidates; security breaches or failure to protect private personal information; attracting and retaining key personnel; and the Companys ability to manage growth.

These and other important factors discussed under the caption Risk factors in the Companys Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 9, 2022 and its other reports filed with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While the Company may elect to update such forward-looking statements at some point in the future, it disclaims any obligation to do so, even if subsequent events cause its views to change. These forward-looking statements should not be relied upon as representing the Companys views as of any date subsequent to the date of this press release.

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Frequency Therapeutics Completes Enrollment of Phase 2b Study of FX-322 for the Treatment of Sensorineural Hearing Loss - Business Wire

- Company will present five posters discussing REACT and chronic kidney disease during the ASN Annual Meeting, November 3 - 6, 2022

- Investor and analyst scientific briefing to be hosted in Orlando, FL on November 3, 2022

WINSTON-SALEM, N.C., Oct. 14, 2022 (GLOBE NEWSWIRE) -- ProKidney Corp. (Nasdaq: PROK) (ProKidney), a leading late clinical-stage cellular therapeutics company focused on chronic kidney disease (CKD), today announced that the Company will present five posters on REACT and CKD at the upcoming American Society of Nephrologys (ASN) Kidney Week being held November 3-6, 2022, in Orlando, FL.

Details for the poster presentations are as follows:

Session Title: CKD: Epidemiology, Risk Factors, Prevention I [PO2201-1]

Session Date, Time: November 3, 2022, from 10:00 AM to 12:00 PM

Poster Board #: TH-PO887

Session Title : Genetics, Development, Regeneration [PO0500]

Session Date, Time: November 4, 2022, from 10:00 AM to 12:00 PM

Poster Board #: FR-PO394

Session Title: CKD: Epidemiology, Risk Factors, Prevention II [PO2201-2]

Session Date, Time: November 4, 2022, from 10:00 AM to 12:00 PM

Poster Board #: FR-PO905

Abstracts can be accessed online at https://www.asn-online.org/education/kidneyweek/.

ProKidney also announced today that it will host an investor and analyst scientific briefing, followed by a reception, the evening of November 3, 2022, in Orlando, FL. To register for the event, or for additional information, please contact Dr. Glenn Schulman at glenn.schulman@prokidney.com. Following the event, a copy of the presentation slides will be available on the Companys website at https://investors.prokidney.com/news-events/events-and-presentations.

About ProKidney

ProKidney, a pioneer in the treatment of CKD through innovations in cellular therapy, was founded in 2015 after a decade of research. ProKidneys lead product candidate, REACT (Renal Autologous Cell Therapy), is a first-of-its-kind, patented, autologous cellular therapy with the potential to not only slow and stabilize the progression of CKD, but in some cases potentially drive meaningful improvement in kidney function. Late-stage CKD patients, Stage 3b - 4, is a key target population for REACT therapy. REACT has received Regenerative Medicine Advanced Therapy (RMAT) designation, as well as FDA and EMA guidance, supporting its ongoing Phase 3 clinical program that launched in January 2022. For more information, visit http://www.prokidney.com.

About CKD

There are no therapies that effectively reverse late-stage CKD. CKD is a serious diagnosis with significant morbidity and mortality. Notably, the 5-year mortality of newly diagnosed Stage 4 CKD is higher than that of newly diagnosed non-metastatic cancer. CKD most often presents as a progressive decline in kidney function, ultimately resulting in the failure of the kidneys and the need for renal replacement therapy, such as hemodialysis or kidney transplant. One in three Americans is at risk for CKD which currently affects approximately 75 million people in the United States and Europe and over 400 million across Asia. CKD is among the largest single expenses incurred by the U.S. health care system.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. ProKidneys actual results may differ from its expectations, estimates and projections and consequently, you should not rely on these forward-looking statements as predictions of future events. Words such as expect, estimate, project, budget, forecast, anticipate, intend, plan, may, will, could, should, believes, predicts, potential, continue, and similar expressions (or the negative versions of such words or expressions) are intended to identify such forward-looking statements. These forward-looking statements include, without limitation, the combined companys expectations with respect to financial results, future performance, development and commercialization of products, if approved, the potential benefits and impact of the combined companys products, if approved, potential regulatory approvals, anticipated financial impacts and other effects of the business combination on the combined companys business, and the size and potential growth of current or future markets for the combined companys products, if approved. Most of these factors are outside of the combined companys control and are difficult to predict. Factors that may cause such differences include, but are not limited to: the inability to maintain the listing of the combined companys Class A ordinary shares on the Nasdaq following the business combination; the inability to implement business plans, forecasts, and other expectations and to recognize the anticipated benefits of the business combination or identify and realize additional opportunities, which may be affected by, among other things, competition and the ability of the combined company to grow and manage growth profitably and retain its key employees; the risk of downturns and a changing regulatory landscape in the highly competitive biotechnology industry; the inability of the combined company to raise financing in the future; the inability of the combined company to obtain and maintain regulatory clearance or approval for its products, and any related restrictions and limitations of any cleared or approved product; the inability of the combined company to identify, in-license or acquire additional technology; the inability of combined company to compete with other companies currently marketing or engaged in the biologics market and in the area of treatment of kidney diseases; the size and growth potential of the markets for the combined companys products, if approved, and its ability to serve those markets, either alone or in partnership with others; the combined companys estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the combined companys financial performance; the combined companys intellectual property rights; uncertainties inherent in cell therapy research and development, including the actual time it takes to initiate and complete clinical studies and the timing and content of decisions made by regulatory authorities; the impact of COVID-19 or geo-political conflict such as the war in Ukraine on the combined companys business; and other risks and uncertainties indicated from time to time in the proxy statement relating to the business combination, including those under Risk Factors therein, and in the combined companys other filings with the Securities and Exchange Commission. The combined company cautions readers that the foregoing list of factors is not exclusive and cautions readers not to place undue reliance upon any forward-looking statements, which speak only as of the date made. The combined company does not undertake or accept any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based.

Contact Information

Investors & Media

Glenn Schulman, PharmD, MPH

SVP, Investor Relations

glenn.schulman@prokidney.com

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ProKidney Announces Multiple Abstracts Selected for Presentation at the American Society of ... - The Bakersfield Californian