Category Archives: Gene Medicine

Gosselies, Belgium, 12 October 2021, 7:00 am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today announces it has appointed key experts to a Scientific Advisory Board (SAB).

Bone Therapeutics has appointed the members of this SAB specifically to provide additional expert guidance on the development of Bone Therapeutics novel, next generation induced pluripotent stem cell-derived mesenchymal stromal cell (iMSC) platform. This iMSC platform will be used to develop cell and gene therapy products that have strong anti-inflammatory and immunomodulatory properties, for the treatment of acute life-threatening unmet medical diseases.

Bone Therapeutics has appointed its SAB with world-recognized scientists and clinicians in the cell and gene therapy field. Each SAB member has been selected having demonstrated leadership roles in the clinical development of engineered cell and gene therapy for specific acute unmet medical conditions. These specific conditions include graft vs host disease, acute respiratory distress syndrome, sepsis, and trauma, as well as orthopedic conditions including osteoarthritis.

Bone Therapeutics is developing a next generation iMSC platform that has the potential to develop transformative cell and gene therapies for patients suffering from a range of life-threatening unmet medical diseases. Given the therapeutic potential of this platform and to deliver this platform to an operational state as quickly as possible, Bone Therapeutics has brought together a group of world-leading experts to support its development, said Tony Ting, PhD, Chief Scientific Officer, Bone Therapeutics. These thought leaders have been selected to bring a wealth of specific experience in the clinical development of cell and gene therapies. The input from this SAB will be critical as Bone Therapeutics develops its next-generation iMSC products for acute inflammatory diseases.

Given the therapeutic potential of the iMSC platform that Bone Therapeutics is developing, the invitation to chair and help form this scientific advisory board was too tempting to decline, said Massimo Dominici, MD, chair, Bone Therapeutics Scientific Advisory Board. The blend in expertise of this scientific advisory board will be able to provide key advice and consultancy to Bone Therapeutics and will make key contributions to ensure the development of the iMSC platform to reach patients of acute life-threatening unmet medical diseases as quickly as possible.

The Bone Therapeutics Scientific Advisory Board are as follows:

Massimo Dominici, MD, (Chair) - Full Professor of Medical Oncology and Director of the Division of Medical Oncology and of the Program of Cellular Therapy and Immuno-oncology at the University Hospital of Modena and Reggio Emilia (Italy). Also a member of the World Health Organization (WHO) Expert Advisory Panel on The International Pharmacopoeia and Pharmaceutical Preparations serving the INN Expert Group. Since 2016, the Director of the Residency School in Medical Oncology, since 2005, head of the Laboratory of Cellular Therapies at the University Hospital of Modena and Reggio Emilia (Italy). Scientific founder of the university start-up Rigenerand since 2009. Co-founder and coordinator of the Mirandola Science & Technology Park. Co-founder of the Forum of Italian Researcher on MSC (FIRST), board member of JACIE, WBMT and scientific advisor for the Italian Minister of Health. President of ISCT 2014-2016, Emeritus Member of ISCT and now Member of the ISCT Strategic Advisory Council. From June 2014 until May 2020 Chair of the ISCT Presidential taskforce on unproven cell and gene therapies.

Frank Barry, PhD, Professor of Cellular Therapy at the Regenerative Medicine Institute (REMEDI), National University of Ireland Galway and Visiting Scientist at the Schroeder Arthritis Institute in Toronto. He has made key contributions to the fields of tissue engineering and regenerative medicine by developing innovative and successful cellular therapies for tissue repair, joint injury and arthritic disease. By undertaking a large body of basic and translational research, he has contributed to the industrys current understanding of the phenotypic attributes of mesenchymal stromal cells that make them attractive candidates for advanced therapeutics. He has also contributed to the development of methods for automated, efficient and scalable cell expansion for GMP application and has been a leader in the development of clinical protocols for patient testing. He is the Coordinator of the ADIPOA2 clinical trial to test the efficacy of stromal cell delivery as a treatment for osteoarthritis. Frank Barry has received the Marshall Urist Award for excellence in tissue regeneration research from the Orthopaedic Research Society. Recently elected as a Member of the Royal Irish Academy.

Robert Deans, PhD, CSO at Synthego, a genome engineering company automating a new era of cell and gene therapeutics. Previously CTO at BlueRock Therapeutics, creating iPSC based allogeneic cell therapeutics by harnessing pluripotent stem cell biology and gene editing tools and founding CSO at Rubius Therapeutics, developing a platform of novel enucleated cell therapeutics based on genetic engineering and expansion of hematopoietic progenitors to mature red cells. Dr. Deans has more than 30 years of experience in adult stem cell therapeutics which includes HSC gene therapy and commercialization of progenitor cell therapeutics from bone marrow. Richard Maziarz, MD, has been involved in clinical investigation and translational research, for over 30 years, beginning with research and clinical training at the Dana-Farber Cancer Institute and the Brigham & Womens Hospital and continuing in 1991 when he moved to Oregon Health & Science University (OHSU) to develop a transplantation immunology program and served as the medical director of the adult OHSU stem cell transplant program since 1994. His research involved the immunology of transplantation or its complications, particularly in studying the immunopathophysiology of GVHD. He has served as principal investigator or co-investigator on over 100 clinical trials including multiple initiatives sponsored by numerous national transplant organizations including SWOG, CIBMTR, ISCT, NMDP and BMT CTN. Within the BMT CTN, he serves on the Steering committee, chaired the Regimen Related Toxicity Committee, was a member of the GVHD Committee and served as the principal investigator for the BMT CTN on the first multicenter, stem cell transplant trial for patients with advanced chronic lymphocytic leukemia (BMT CTN 0804).

Patricia Rocco, MD, PhD, Full Professor at the Federal University of Rio de Janeiro, and heads the Laboratory of Pulmonary Investigation. Elected Member of the National Academy of Medicine in Brazil and Brazilian Academy of Science. Past Vice-President of ISCT for the South and Central America regions. Authored and co-authored more than 380 peer-reviewed publications and 120 book chapters. She is the President of the Brazilian Society of Physiology (2021-2022). Her research activities focus mainly on the development of new therapies for lung diseases.

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a diversified portfolio of cell therapies at different stages ranging from pre-clinical programs in immunomodulation to mid stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell and gene therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available at http://www.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerLieve Creten, Chief Financial Officer ad interimTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics appoints Scientific Advisory Board for iMSC cell and gene therapy platform development - GlobeNewswire

The birth of the first IVF baby, Louise Brown, in 1978 provoked a media frenzy. In comparison, a little girl named Aurea born by IVF in May 2020 went almost unnoticed. Yet she represents a significant first in assisted reproduction too, for the embryo from which she grew was selected from others based on polygenic screening before implantation, to optimise her health prospects.

For both scientific and ethical reasons, this new type of genetic screening is highly controversial. The nonprofit California-based organisation the Center for Genetics and Society (CGS) has called its use here a considerable reach by the assisted-reproduction industry in the direction of techno-eugenics.

The polygenic screening for Aurea was provided by a New Jersey-based company called Genomic Prediction. The gene-sequencing company Orchid Biosciences in California now also offers an embryo-screening package that assesses risks for common diseases such as heart disease, diabetes and schizophrenia.

Genetic screening of IVF embryos for health reasons, known as preimplantation genetic diagnosis or PGD, is not new in itself. In the UK, it is permitted by the Human Fertilisation & Embryology Authority (HFEA), which regulates assisted conception technologies, to look for specific gene variants associated with around 500 diseases, including cystic fibrosis and Tay-Sachs disease.

The diseases conventionally screened with PGD are mostly caused by a mutation in only a single gene. They can be nasty but are typically rare. In contrast, most common health problems, such as heart diseases or type 2 diabetes, are polygenic: caused by complex interactions among several, often many, genes. Even if particular gene variants are known to increase risk, as for example with the BRCA1/2 variants associated with breast cancer, such links are probabilistic: theres no guarantee that people with that variant will get the disease or that those who lack it will not.

Thats simply how most genes work: in complex, interconnected and often poorly understood ways, so that the gene variants an individual carries dont guarantee which traits they will develop. And environmental factors such as upbringing and diet, as well as unpredictable quirks of embryo development, also have a role. Were products of (genetic) nature, nurture, chance and an interplay between all three.

Yet the availability today of genetic data for many thousands of individuals, thanks to the plummeting costs of genome sequencing and the popularity of genomic profiling companies such as 23AndMe and Orchid, has transformed our understanding of how genes relate to traits. The technique known as a genome-wide association study (GWAS) can sift through vast databanks to look for statistical associations between an individuals gene variants and pretty much any trait we choose. Such studies have found that often substantial amounts of the differences between individuals can be linked to different variants (alleles) of many genes. Each gene might contribute only a tiny effect too small to be apparent without plenty of data - but added together, the influence of the genes can be significant.

So someones genetic profile the variants in their personal genome can be used to make predictions about, say, how likely they are to develop heart disease in later life. They can be assigned a so-called polygenic risk score (PRS) for that condition. Aureas embryo was chosen because of low PRSs for heart disease, diabetes and cancer. PRSs can be used to predict other things too, such as a childs IQ and educational attainment.

But such predictions are probabilistic, both because we cant say exactly how our genes will play out in influencing that trait and because genes arent the only influence anyway. So theres nothing inevitable or deterministic about a PRS. An individual with a high PRS for skin cancer might never develop it, while someone who scores low might do so. Someone with a genetic profile that predicts a modest IQ might turn out to be brilliant.

This is one reason why using PRSs in embryo screening which is legal and largely unregulated in the US is controversial. Unlike single-gene diseases, where the health outcome can be almost certain, its not clear how much faith we can put in predictions for polygenic traits. Yet we make choices based on probabilities all the time. We cant be sure that a particular school will be best for our childs education, but we may decide it will improve the chances of a good outcome. If one embryo has low PRSs for common diseases and another has high ones, doesnt it make sense to pick the first? Aureas father, North Carolina neurologist Rafal Smigrodzki, has argued that part of a parents duty is to make sure to prevent disease in their child. Polygenic testing, he says, is just another way of doing that.

Embryo screening is already used for BRCA1 and 2, even though it is by no means certain that women who carry them will develop breast cancer. Advocates of PRS screening say that it merely improves the risk assessment by widening the genetic factors considered. Most families with a history of breast cancer do not carry the BRCA allele and would benefit from polygenic screening, says Genomic Predictions founder, Stephen Hsu, a professor of physics at Michigan State University. The potential public health benefits are huge. Ethics philosophers Sarah Munday and Julian Savulescu have argued in favour of allowing polygenic screening for any trait that can be shown to be correlated with a greater chance of a life with more well-being.

Theres a scientific basis to the concept [of PRSs] and its a type of genetic assessment that has a future in medicine, says bioethicist Vardit Ravitsky of the University of Montreal. Yet most regulators and many experts feel that there is not yet any justification for using them to try to improve the health outcomes of IVF children. Its not seen as ready for primetime use, says Ravitsky. Its still at a research stage. So when you start jumping straight into implementation, especially in a reproductive context, youre in a minefield. An article in the New England Journal of Medicine in July pointed out that benefits of PRS embryo selection are likely to be very small, all the more so for people not of European heritage, for whom genomic data are less extensive and so less reliable for prediction.

If PRS gives you the power to reduce your offsprings lifetime risk of type 2 diabetes from 30% to 27%, is that worth the time, money, and emotional investment? asks bioethicist Hank Greely of Stanford University in California. And to whom? Thats very different, he says, from the confidence with which single-gene diseases can be screened and avoided.

And once such screening methods are permitted, where does it stop? Already, American couples can screen embryos for gender, complexion and eye colour. Whats to stop a company offering to screen for a non-disease trait such as height or intelligence? Theres no reason to think polygenic embryo screening will end with conditions like heart disease and diabetes, says Katie Hasson, associate director of the CGS. Screening for schizophrenia and other mental illnesses is already on offer. These directly echo eugenic efforts to eliminate feeble-mindedness. We are talking about deciding who should be born based on good and bad genes.

Genomic Prediction has previously offered to screen for gene variants associated with intellectual disability, but Hsu stresses that now the company only offers the service for serious disease risks. We decided that traits like height and cognitive ability are too controversial and detract from our ability to help families reduce disease risk, he says.

Its not clear that screening for such non-disease traits would work anyway. I think the things that parents are most interested in, like intelligence, sports and musical ability, will have extremely small to nonexistent convincing PRS results, says Greely. A study in 2019 suggested that using polygenic screening to select embryos for height and IQ would be likely to make only a tiny difference on average and theres a fair chance you wouldnt end up picking the best embryo.

So what should be permitted? Hsu says: We hope that in the future, society as a whole, perhaps on a nation-by-nation basis, will reach a consensus on which non-disease traits are acceptable for embryo screening. Some have objected to his implication that, say, welfare dependence or criminality are in the genes. Hsu has also attracted controversy because of his comments on whether there are genetically based differences in IQ between racial groups, although he says he is agnostic on the issue. An outcry about his remarks on such matters compelled him to resign in 2020 as his universitys senior vice-president of research and innovation.

Hsu was also one of the scientists suggested by Dominic Cummings to run the UKs new Advanced Research and Invention Agency. In 2014, Cummings blogged about how the NHS should cover the cost of selecting embryos for IQ; in 2019, he was pictured outside 10 Downing Street with Hsu.

To avoid any Gattaca-style genetic stratification of society, Hsu has expressed the hope that progressive governments will make this procedure free for everyone. But Hasson believes that this wouldnt solve the problems of inequality that such techniques could exacerbate. Even if PRSs for smartness, say, have little real predictive value, she says that belief in genomic predictions can itself be a driver of intense inequalities in society by reinforcing ideas of genetic determinism. Families that invest their money, time and hopes in this kind of screening and selection will have children they believe are genetically superior and those children will be treated as superior by their parents, care-givers and educators.

Social pressure could make it hard to resist polygenic screening if its on offer in our hyper-competitive societies. Once you do IVF, you feel pressure to use any add-on service or test that the clinic offers you, says Ravitsky. Look at what happens today when a woman declines prenatal screening or amniocentesis. Many women feel judged, not just by peers but by healthcare providers. The idea that its all about autonomy of choice can be an illusion, she says.

Even if PRSs have little real value in forecasting the prospects of a child, evidently a market exists for them. In countries such as the US where assisted conception is weakly regulated, companies can make unrealistic and exploitative promises. Couples might even elect to have a child via IVF specifically to avail themselves of such opportunities. Its a gruelling process that carries risks in itself, but women might feel compelled to use it, even though Ravitsky thinks that allowing someone to do so for this reason alone would be borderline malpractice.

Yet the genie is out of the bottle. I believe that polygenic screening will become very common in the near future, Hsu says. Reasonable people will wonder why the technology was ever controversial at all, just as in the case of IVF. The HFEA is still considering its implications, says its chief executive, Peter Thompson, who stresses that it is currently illegal in the UK. Even if there were more scientific consensus about the value of PRSs, he adds, there is an important distinction between embryo selection to avoid serious harm and for so-called enhancement, like greater intelligence. The latter would represent a fundamental public policy shift. It raises a range of ethical concerns and could only be contemplated if it has the backing of society more generally, he says.

We urgently need public and policy conversations about polygenic embryo screening, says Hasson. Finding the right balance between autonomy and social responsibility is the fundamental dilemma of liberal democracies. We let people spend their money, and make decisions powerfully affecting their kids, on far more clearly bogus information than PRS, says Greely.

As a society, were very far from knowing how we want to use these potential technologies, says Ravitsky, but, she adds, we are already living in the grey zone.

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Polygenic screening of embryos is here, but is it ethical? - The Guardian

The Brain & Behavior Research Foundation named Justin Wolter, PhD, postdoc in the Neuroscience Research Center, as a recipient of the Young Investigator Award.

Justin Wolter, PhD, a postdoctoral researcher in the labs of Jason Stein, PhD, and Mark Zylka, PhD, at the UNC Neuroscience Research Center, the UNC Department of Genetics, and the UNC Department of Cell Biology and Physiology, was named a recipient of the 2021 Young Instigator Award by the Brain & Behavior Research Foundation (BBRF). The award is for $70,000 over two years.

In his research at the UNC School of Medicine, Wolter aims to understand the molecular and cellular mechanisms of neurodevelopmental diseases. With the BBRF award, he will establish a resource to systematically identify genetic interactions between high-risk autism genes and common genetic variation. This project will build upon work in which Wolter established a cell culture-based approach to conduct genome wide association studies in primary human neural progenitor cells.

Wolter will establish a pilot library of genetically diverse induced pluripotent stem cell (iPSC) lines to explore how common and rare genetic variation interact to influence risk and resilience in a genetically defined subtype of autism.

In 2020, Wolter was first author of a Nature paper from the Zylka lab showing how to use the gene-editing technology CRISPR-Cas9 as part of a potential gene therapy approach to treating Angelman syndrome, an autism spectrum disorder.

Initiated in 1987, the BBRF Young Investigator Grant program provides support for the most promising young scientists conducting neurobiological and psychiatric research. This program facilitates innovative research through support of early-career basic, translational and clinical investigators.

This year, the Foundations Scientific Council, led by Herbert Pardes, MD, and comprised of 176 world-renowned scientists with expertise in every area of brain research, reviewed more than 780 applications and selected the 150 meritorious research projects. Many of the Young Investigator grantees are pursuing basic research projects. Others are specifically focusing on new ideas for therapies, diagnostic tools, and technologies. These research projects will provide future insights and advances that will help move the fields of psychiatry and neuroscience forward.

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Wolter Earns Young Investigator Award | Newsroom - UNC Health and UNC School of Medicine

PHILADELPHIAReferred to as a Beacon of Hope, Penn Medicines new Pavilion, one of the largest hospital projects underway in the United States, opens to staff, patients, and visitors this month.

The 17-story bronze-colored building rises on Penn Medicines West Philadelphia campus, housing 504 private patient rooms and 47 operating rooms, as an expanded footprint of the Hospital of the University of Pennsylvania (HUP). The $1.6 billion facility is poised to serve as the launch pad for Penn Medicines next generation of pioneering advances in patient care.

From the design and construction process to staff training, this state-of-the-art hospital has been home to a variety of innovative approaches that are mapping the future of healthcare. Highlights include:

The uncertainties experienced throughout the COVID-19 pandemic have emphasized the need for designing a hospital that could adapt with rapidly changing science, medicine, and patient care. The Pavilion not only has enhanced infection control capabilities, but it was designed to help accelerate bench-to-bedside research, and it incorporates state-of-the-art technologies for caregivers, such as rooms with telemedicine functionality to allow for remote monitoring and consultations.

In the center of a vibrant clinical and research campus, the Pavilion is a centerpiece of Penn Medicines world-class expertise in bold approaches to treating diseases of all kinds, from cell and gene therapies to specialized cardiac surgeries.

The Pavilion is designed to transform the patient and family experiencetheir experience starts with a reassuring welcome in the lobby, and extends through comfortable wards, private rooms, and spaces for caregivers.

While the Pavilion is a place that promotes healing through patient care, it also creates a restorative environment by providing calming art and nutritious food for patients, visitors, and staff.

Ten thousand employees completed training before the Pavilion opens its doors for patients. This comprehensive training program is built on years of employee feedback, testing, and participation.

Sustainability has been a key aspect of the Pavilions construction and design from the beginning, making it on track toward receiving a Leadership in Energy and Environment Design (LEED) Gold Certificationa globally recognized symbol that promotes achievement in sustainable design and construction.

Penn Medicineis one of the worlds leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of theRaymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nations first medical school) and theUniversity of Pennsylvania Health System, which together form a $8.9 billion enterprise.

The Perelman School of Medicine has been ranked among the top medical schools in the United States for more than 20 years, according toU.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $496 million awarded in the 2020 fiscal year.

The University of Pennsylvania Health Systems patient care facilities include: the Hospital of the University of Pennsylvania and Penn Presbyterian Medical Centerwhich are recognized as one of the nations top Honor Roll hospitals byU.S. News & World ReportChester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; and Pennsylvania Hospital, the nations first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is powered by a talented and dedicated workforce of more than 44,000 people. The organization also has alliances with top community health systems across both Southeastern Pennsylvania and Southern New Jersey, creating more options for patients no matter where they live.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2020, Penn Medicine provided more than $563 million to benefit our community.

Link:
Patient Care on the Precipice of Transformation at Penn Medicine's New Pavilion - pennmedicine.org

SAN DIEGO, Oct. 7, 2021 /PRNewswire/ -- ViaCyte, Inc., an innovator in cellular therapy and regenerative medicine, announced today that it will present at the Alliance for Regenerative Medicine Cell & Gene Meeting on the Mesa on October 14, 2021, at 10:15 a.m. PT in Carlsbad, CA.

Michael Yang, ViaCyte's President and Chief Executive Officer, will give an update on the Company's latest advances in the development of product candidates for long-term treatment of type 1 diabetes, to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications.

"Cell replacement therapy has the potential to address the limitations of current therapies and advance the treatment of diabetes," said Mr. Yang. "Recently, ViaCyte has demonstrated a clinical proof-of-concept that our stem cell-derived islet replacement therapy is a promising approach to effectively produce insulin and regulate blood glucose in patients with type 1 diabetes."

The Cell & Gene Meeting on the Mesa is the sector's foremost annual conference bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures.For more details, visit http://www.meetingonthemesa.com.

About ViaCyte

ViaCyte is a privately held clinical-stage regenerative medicine company developing novel cell replacement therapies based on two major technological advances: cell replacement therapies derived from pluripotent stem cells and medical device systems for cell encapsulation and implantation. ViaCyte has the opportunity to use these technologies to address critical human diseases and disorders that can potentially be treated by replacing lost or malfunctioning cells or proteins. The Company's first product candidates are being developed as potential long-term treatments for patients with type 1 diabetes to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications. To accelerate and expand the Company's efforts, ViaCyte has established collaborative partnerships with leading companies, including CRISPR Therapeutics and W.L. Gore & Associates. ViaCyte is headquartered in San Diego, California. For more information, please visit http://www.viacyte.comand connect with ViaCyte on Twitter, Facebook, and LinkedIn.

SOURCE ViaCyte, Inc.

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ViaCyte to Present at Alliance for Regenerative Medicine Cell & Gene Meeting on the Mesa - PRNewswire

Science is a lot more boring than it is commonly portrayed. Movies often show montages of bespectacled scientists scribbling notes (probably on a chalkboard) before they finally punch the air in delighted revelation. Or maybe they show a huge team of researchers spending years on some scientific problem, and then the protagonist turns a blueprint upside down and says, but could this be it? Everyone is amazed.

The reality of science is far more prosaic. It is years upon years of hard graft, dead ends, worrying about funding, conferences, more dead ends, more hard graft, and awholelot of collaboration. Science is less about eureka moments and lone geniuses and more about standing on the shoulders of giants. But occasionally, a development bucks the trend, giving at least some validation to the Hollywood tropes.

One example is in the truly revolutionary gene-editing technology known as CRISPR. The tool is incredible not just for what it can do and how it might change human life, but also for its origin story a tale of a game-changing discovery, a eureka moment, and research conducted for researchs sake.

The story starts in 1987 when a Japanese research team headed by Yoshizumi Ishino was researching the microbe E. coli. They wanted to explore a peculiar gene called iap. This mysterious gene was unique, consisting of blocks of five identical segments of DNA divided by unique spacer DNA. But because this was the 1980s and the technology wasnt sophisticated yet, the Osaka team didnt really know what to make of the observations, or what to do with them.

Fifteen years later in the Netherlands, a team headed by Francisco Mojica and Ruud Jansen of Utrecht University renamed these sandwiches of iap to CRISPR, which means clustered regularly interspaced short palindromic repeats. What Mojica, Jansen et al. discovered was remarkable: These genes encoded enzymes that couldcut DNA. Still, no one knew why this happened, and the implications of this werent fully appreciated.

What they discovered is that the CRISPR system could be reprogrammed to cut and paste not only virus DNA, but also whatever isolated DNA they wanted.

Three years later, Eugene Koonin at the National Center for Biotechnology Information, noticed that these unique DNA bits in the spacers looked remarkably like viruses. And so, Koonin theorized that certain microbes were using CRISPR as a defense mechanism. It was a bacterial immune system. He suggested that bacteria used CRISPR (and their cas enzymes) to take fragments of invasive viruses and then paste them into their own cut DNA, where they acted as a kind of bacterial vaccination against future viruses, or like an immune-system memory.

It was left for microbiologist Rodolphe Barrangou to prove Koonin right. CRISPR really was cutting and pasting DNA.

The implications of this were rather lost on both Barrangou and the microbiologist community. Barrangou himself used (and monetized) this technology to make virus-resistant bacteria for his yogurt-making employer Danisco. But on the other side of the country, at the University of Berkeley, these findings were being read by two people who would transform CRISPR technology: Jennifer Doudna and Emmanuelle Charpentier.

Doudna and Charpentier were experts in the field of RNA the blueprints created by DNA that act as the messenger required to encode all the proteins of life. What they discovered is that the CRISPR system could be reprogrammed to cut and paste not only virus DNA, but also whatever isolated DNA they wanted. They published their findings in a now-famous2012Sciencearticle.

But what does reprogram actually mean? First, we have to understand that CRISPR not only cuts and pastes virus DNA into its own DNA (as an immune-memory system or look-up table), but also uses this information to cut up future invader viruses, which prevents them from replicating. It does this by releasing RNA matching the virus DNA (which it has stored)along withits own cas enzyme. If these two find any invader virus DNA, they latch on, and the cas enzyme cuts it in two. Its an incredibly clever process.

This finding produced the eureka moment: Oh my gosh, this could be a tool! Doudna recalled. To make that tool, they simply needed to attach this casenzyme to an RNA of their own choosing, so that the enzyme would find and cut the matching DNA to that RNA. Its sort of like a microbial find and cut function. Whats more, they could then induce a cell to stitch genes to fill the gap a type of find and replace function.

The implications of what Doudna and Charpentier discovered have opened new and unprecedented opportunities. Since their original 2012 paper,an increasing number of companiesand research operations have been conjuring up exciting ways to apply CRISPR technology. Not only does it have huge application in biomedical fields, such as targeting the protein dystrophin responsible for many types of muscular dystrophy, but it also could transform agriculture, energy, and evenmammoth rewilding.

As with any new technology, there are dangers and ethical questions surrounding the use of CRISPR, especially concerning the prospect of creating designer babies. In 2018, the issue stepped out of the theoretical realm when the Chinese scientist He Jiankui edited human embryos for the first time in history, in an attempt to make the babies resistant to the HIV virus. (He was sentenced to three years in prison.) Arguably, these are normal calibration issues that society must deal with when faced with a revolutionary technology.

Whats doubly great about CRISPR is the story behind it. Across decades and continents, the story has involved accident, eureka, and out-of-the-box thinking. But its important to note that the research was done for its own sake. It was conducted to study E. coli, to examine bacterial immune systems, and to develop stronger yoghurt cultures, all while, in the words of Jennifer Doudna, not trying to get to a particular goal, except understanding. The research ultimately accomplished much more than that.

This article was originally published on our sister site, Big Think. Read the original article here.

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CRISPR is revolutionizing medicine its origin story is pretty incredible, too - Freethink

The newly-created Longevity Science Foundation aims to extend the human lifespan to more than 120 years by channeling over 860M ($1B) into early-stage geroscience research in the next decade. Experts say thats a worthy if complex goal.

Based in Zug, Switzerland, the Longevity Science Foundation will prioritize four areas of research: personalized medicine, therapeutics, artificial intelligence (AI) and predictive diagnostics. The foundation accepts applications from any organization with a focus on funding early-stage academic research into the process of aging, known as geroscience. The mission for the projects it funds is to make a difference in peoples lives within five years.

The foundation has already raised an undisclosed amount to fund an initial round of projects and will continue raising the rest of its 860M ($1B) target over the next 10 years. Its setup expenses were covered by founders of LongeVC, a Swiss venture capital fund focused on the anti-aging niche. The Longevity Science Foundation board includes members of LongeVC in addition to research organizations including the National University of Singapore and Human Longevity in the US.

The goals of the foundation are ambitious, if the past decade or so in European biotech, fraught with delays and under-fulfilled promise in geroscience, is any indication. At the same time, they fit neatly into industry projections for the next decade, where sectors such as AI, gene therapy, and personalized medicine feature prominently.

The foundation is bent on taking head-on some of the major financing bottlenecks of translating geroscience research from the lab into clinical-stage anti-aging strategies, which is widely seen as a major boost for the thorny science of aging.

After spending five years in this sector it is so exciting to see substantial capital finally coming in to move the science forward, Greg Bailey, the CEO of the Dublin-based geroscience biotech Juvenescence, told me. This amount of capital and the quality of the individuals that are involved should be transformative to the trajectory of the science to modify aging.

The niche as a whole has had a bumpy ride in recent years. One example is the US company Unity Biotechnology, which is still reeling from a phase II clinical trial failure last year of its small molecule drug designed to treat osteoarthritis.

Unitys drug targeted the accumulation of senescent cells, which stop being able to perform their function with age and can drag entire organs and body systems down with them. The phase II setback called into question the viability of targeting senescent cells, though in July this year, the company touted promising phase I results of a similar drug for the treatment of age-related blindness conditions.

Investor interest in geroscience research has more broadly been hampered by the lack of a unified theory that explains all the different symptoms and conditions that characterize aging. Despite numerous attempts to identify biomarkers that can predict aging in recent years, no clear answer exists even to the question of whether aging is a disease or a catch-all term that includes a number of different processes. Additionally, aging itself isnt seen as an indication by major regulators including the EMA and FDA.

Other approaches to slowing down or reversing aging include repairing the chromosomes, which carry the genetic material of the cells; fixing the mitochondria, which are vital for cell metabolism; and replacing old tissue, especially by virtue of stem cell therapies. All these geroscience approaches carry potential risks as well as limitless promise.

Weve pivoted from a mitochondria-focused approach to cellular reprogramming, a powerful rejuvenation paradigm for cells, said Daniel Ives, the CEO of the UK biotech Shift Bioscience.

Cellular reprogramming is currently a goldilocks rejuvenation approach: too little and you have rejuvenation, too much and you risk cancer. Based on a novel application of machine learning, weve identified an opportunity to tame cellular reprogramming and safely reset cells and tissues back to a youthful state.

The Longevity Science Foundation aims to help scientists take on these issues by providing funds to early-stage researchers with no strings attached. It places a special emphasis on addressing inequalities: both in terms of patient access to cutting-edge treatments, and the access of scientists from diverse parts of the world and backgrounds to research funds.

We know a key barrier to these advancements and one of the most significant challenges facing longevity research today is the lack of transparent, equity-free funding for early-stage discovery and research, Garri Zmudze, a life sciences angel investor and the Executive Coordinator of the foundation, told me.

That is why, in all projects awarded funding by the Foundation, the [intellectual property] will remain the property of its respective researchers and owners. Donors will not, at this time, receive a financial stake in the research or IP.

The field, alongside many other medical niches, has seen an increase of investments in recent years. It also received a boost from a much-publicized trial of metformin, a diabetes drug that is also believed to have anti-aging properties. The TAME trial, which also aims to help discover reliable biomarkers for aging, is currently preparing to launch and will run for six years.

Many are hopeful that the Longevity Science Foundation, or at least geroscience research that comes out of the projects it awards, will help provide the next much-needed breakthrough.

In the short term, market approval of the first drug with an anti-aging or rejuvenation mechanism for an age-linked disease would mark a major milestone for the field, Ives said. In the medium term, the development of a safe but powerful rejuvenative drug would catalyze the regulatory change required to enable the approval of drugs with a full anti-aging or rejuvenation label.

Cover image via Anastasiia Slynko. Body text image via Shutterstock

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Longevity Foundation to Fund Geroscience Research with 860M - Labiotech.eu

Does the knowledge of nerve impulses which can perceive temperature and pressure when initiated help to treat pain?

The story so far: The 2021 Nobel Prize in Physiology or Medicine was jointly awarded to David Julius, 66, at the University of California, San Francisco, and Ardem Patapoutian, 54, at Scripps Research, La Jolla, California, for their discoveries of receptors for temperature and touch.

Editorial | Sensing heat: On 2021 Nobel Prize in Physiology or Medicine

What is the significance of their work?

The two researchers discovered the molecular mechanism by which our body senses temperature and touch. Being able to do this opens the field for a lot of practical chemistry whereby individual cells and pathways can be tweaked, suppressed or activated to quell pain or sensation. How the body senses external stimuli is among the oldest excursions of natural philosophy. Entire schools of philosophy were based on speculating how the senses influenced the nature of the reality we perceive. Only when physiology developed as an independent discipline and anatomy came into its own did it become widely accepted that specific sensations were the result of different categories of nerves getting stimulated. Thus, a caress or a punch induces cells in our bodies to react differently and convert into specific patterns of electrical stimulation that is then conveyed via the nerves to the central nervous system. Since the Nobel Prizes came to be, at least three of them were for establishing key principles for how sensations travelled along skin and muscle sensory nerve fibres. Much like the length, thickness, material and incident force on their strings elicit specific tones out of a guitar or a piano, there are specific nerve fibre types that in tandem create a response to touch, heat and proprioception, or the sense of our bodys movement and position in space. However, the prominence of molecular biology means that physiology wanted to go a level deeper and find out what specific proteins and which genes are responsible in this symphony of the nerves.

What is the contribution of David Julius towards this?

Capsaicin (8-methyl-N-vanillyl-6-nonenamide), the active component of chili peppers, generates the burning sensation when eating spicy food. Studies on capsaicin showed that when it acted on sensory nerves it induced ionic currents, or the gush of charged particles along a membrane. In the late 1990s, Professor Julius pursued a project to identify a nerve receptor for capsaicin. He thought that understanding the action of capsaicin could provide insights into how the body sensed pain. He and his team went about this by looking for a gene that could induce a response to capsaicin in cells that usually wouldnt react to it. They found one in a novel ion channel protein, later called TRPV1, where TRP stands for transient receptor potential, and VR1 is vanilloid receptor1. They were part of a super family of TRP and it was found that TRPV1 was activated when temperatures were greater than 40 degrees Celsius, which is close to the bodys pain threshold. Several other TRP channels were found, and this ion channel could be activated by various chemical substances, as well as by cold and heat in a way that differs between mammalian species.

What did Ardem Patapoutian find?

Growing up in Beirut as an Armenian, during the Lebanese Civil War, Patapoutian has related stories of being captured by militants at university, before he moved to the United States. Patapoutian and his colleagues were working on how pressure and force affected cells. Following an approach similar to that of Professor Julius, they identified 72 potential genes that could encode an ion channel receptor and trigger sensitivity to mechanical force, and it emerged that one of them coded for a novel ion channel protein, called Piezo1. Via Piezo1, a second gene was discovered and named Piezo2. Sensory neurons were found to express high levels of Piezo2 and further studies firmly established that Piezo1 and Piezo2 are ion channels that are directly activated by the exertion of pressure on cell membranes. The breakthrough by Professor Patapoutian led to a series of papers from his and other groups, demonstrating that the Piezo2 ion channel is essential for the sense of touch. Moreover, Piezo2 was shown to play a key role in proprioception as well as regulate blood pressure, respiration and urinary bladder control. Independently of one another, Professor Julius and Professor Patapoutian used the chemical substance menthol to identify TRPM8, a receptor activated by cold.

What applications do these discoveries have?

Along with the discoveries of specific genes, proteins and pathways, the scientists pioneered experimental methods that allow insight into the structure of these pain and temperature sensors. The challenge for pain relieving drugs is to precisely target regions without causing imbalance in other necessary functions. These scientists work, the Nobel Prize committee said, significantly helped towards reaching that goal.

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Explained | The 2021 Nobel Prize in Physiology or Medicine - The Hindu

Globally, cell and gene therapy products are transforming the treatment of cancers and genetic diseases.

ARLINGTON, Va. (PRWEB) October 07, 2021

Cell and gene therapies are trending as groundbreaking treatments with the potential to actually cure disease rather than simply manage symptoms. As a result, through 2030, the global market for cell and gene therapy is forecast to expand strongly to $29,960 million, up from $3,866 million in 2020, according to a 2021 report by Kalorama Information and editors at the medical research firms new sister publication, Cell and Gene Therapy Business Outlook.

Globally, cell and gene therapy products are transforming the treatment of cancers and genetic diseases, said Bruce Carlson, publisher for Kalorama Information. Additionally, cell and gene therapies are expanding into other areas of medicine including autoimmune diseases, cardiovascular diseases, musculoskeletal disease, dermatological diseases and many others.

Oncology a Key Cell and Gene Therapy SegmentAmong the expanding areas of focus for cell and gene therapies, oncology remains one of the most intriguing and notable. Treating cancer is difficult and not just because there are over 100 different types of cancer (including lung, breast, brain, blood, prostate and colon cancer), but because it is not a single disease and because all the cells in a single tumor do not behave in the same way. Although most cancers are thought to be derived from a single abnormal cell, by the time a tumor reaches a clinically detectable size, the cancer may contain a diverse population of cells.

Opportunities Amidst ChallengesDespite the challenges, there are opportunities. Cell therapy has been used for years for blood transfusions and hematopoietic stem cell transplants. Advancements in technology have created new options for treatment. The initial development of CAR-T cell therapies focused on treatment for ALL (acute lymphoblastic leukemia), NHL (non-Hodgkin lymphomas), multiple myeloma, and GBM (glioblastoma multiforme). Now in development are products for solid tumors such as pancreatic cancer, ovarian cancer and colorectal cancer. The FDA has even approved three CAR-T cell therapies, including Kymriah, Yescarta, and Tecartus.

Growth Factors in 2021Kalorama Information estimates the global market for cell and gene therapy for oncology reached $1,582 million in 2020 and is expected to climb to $2,744 million in 2021 as the segment exhibits tremendous growth momentum that is expected to continue through 2030. Several factors contributed to strong growth including:

Growth Factors Beyond 2021Pricing Trends In oncology cell and more particularly, gene therapy, the cost of therapies is exceptionally high. From development to manufacturing processes, these therapies require more complex evaluations to determine eligibility for payment. It is anticipated that as more products become available, prices will fall somewhat but it is an expensive process for all participants.

Expected Market Penetration It is anticipated that CAR-T oncology products will continue to produce the lion share of revenues for the oncology cell and gene therapy market. Compound annual growth rate for CAR-T is forecast to be 43% for 2020-2025. Market penetration was slowed in 2020 due to the COVID-19 virus but is expected to gain steadily in 2021-2025. The CAR-T market will overshadow the cell immunotherapy, gene therapy and other cell therapy markets.

More information about Cell and Gene Therapy Business Outlook can be found here: https://kaloramainformation.com/cell-and-gene-therapy-business-outlook/

About Kalorama Information:Kalorama Information, part of Science and Medicine Group, is the leading publisher of market research in healthcare areas, including in vitro diagnostics (IVD), biotechnology, medical devices, and pharmaceuticals. Science and Medicine Group supports companies seeking to commercialize the rapidly changing marketplace at the intersection of science, medicine, and technology. Comprised of industry-leading brands, Science and Medicine Group serves analytical instrument, life science, imaging, and clinical diagnostic companies by helping them create strategies and products to win markets and provide platforms to digitally engage their markets through a variety of innovative solutions. Kalorama Information produces 30 reports a year. The firm offers a Knowledge Center, which provides access to all published reports.

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Cancer is a Key Focus in Cell and Gene Therapy Research - PR Web

The 2021 Nobel prize in physiology or medicine has been awarded to two U.S. scientists who discovered the microscopic secrets behind the human sense of touch.

David Julius, of the University of California San Francisco, received half of the prize for using "capsaicin, a pungent compound from chili peppers that induces a burning sensation, to identify a sensor in the nerve endings of the skin that responds to heat," while Ardem Patapoutian, of the Scripps Research Institute in La Jolla, California, received the other half for using "pressure-sensitive cells to discover a novel class of sensors that respond to mechanical stimuli in the skin and internal organs," the Royal Swedish Academy of Sciences announced Monday (Oct. 4).

Their discoveries "have allowed us to understand how heat, cold and mechanical force can initiate the nerve impulses that allow us to perceive and adapt to the world around us," the Nobel Committee said in a statement. "This knowledge is being used to develop treatments for a wide range of disease conditions, including chronic pain."

Related: 7 revolutionary Nobel Prizes in medicine

The award comes with a prize of 10 million Swedish kronor ($1.15 million) to be shared equally between the two winners.

Beginning in the 1990s, the scientists pieced together the molecular pathways that translate heat and pressure detected on the skin into nerve impulses perceived by the brain. Julius and his colleagues started the work by creating a library of millions of DNA segments containing genes found in sensory nerve cells. By adding the genes one by one to cells that did not normally react to capsaicin, they eventually found that a single gene was responsible for the burning sensation associated with capsaicin. The gene they had discovered gave cells the ability to build a protein called TRPV1, which was activated at temperatures hot enough to be considered painful.

Both Julius and Patapoutian independently went on to use menthol to discover another protein, TPRM8, which was activated by cold temperatures, as well as a number of other proteins that detected a range of different temperatures.

Building on this work, Patapoutian and his colleagues created a library of 72 genes that they suspected encoded blueprints to make receptors for mechanical pressure. By painstakingly deactivating these genes one by one in cells, they discovered that one of the genes produced a protein that spurred cells to produce a tiny electrical signal each time they were prodded. The receptor they had discovered was not only vital for sensing mechanical force, but was also used in various ways to maintain blood vessels, alongside having a proposed role in adjusting the bodys blood pressure.

Soon after that, they found a second protein receptor that was vital in sensing body position and motion, a sense known as proprioception. They named the two receptors Piezo1 and Piezo2, after the Greek word for pressure.

Not only did the discoveries help explain the mechanisms behind sensory experiences like temperature and pressure, but they also opened up a world of possibilities for new drugs targeting the receptors from painkillers to drugs that could alleviate blood pressure across blood vessels and organs.

"While we understood the physiology of the senses, what we didn't understand was how we sensed differences in temperature or pressure," Oscar Marin, director of the MRC Centre for Neurodevelopmental Disorders at Kings College London told The Associated Press. "Knowing how our body senses these changes is fundamental because once we know those molecules, they can be targeted. It's like finding a lock and now we know the precise keys that will be necessary to unlock it."

Joseph Erlanger and Herbert Gasser, who shared the Nobel prize in physiology or medicine in 1944, first discovered specialized nerve cells responsive to both painful and non-painful touch.

Last year's prize went to three scientists for their discovery of hepatitis C, a blood-borne virus that causes chronic liver inflammation. The deadly disease's discovery was a breakthrough that enabled doctors to identify the virus in patients' blood and develop a cure, Live Science previously reported.

Originally published on Live Science.

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Nobel prize in medicine won by US scientists who unlocked the secrets of our sense of touch - Livescience.com