Stem Cell Clinic

Pioneers of cellular reprogramming and cell therapies join bit bio – PRNewswire

CAMBRIDGE, United Kingdom, Oct. 28, 2019 /PRNewswire/ -- bit bio(formerly Elpis Biomed) today announced its founding team, a group of pioneering scientists and business executives at the forefront of stem cell biology, cellular reprogramming and cell therapies. Biotech entrepreneur and scientist Paul Morrill will join as Chief Business Officer; and biotech veteran Florian Schuster will join as CFO/COO.

The company also announced today two additions to its scientific advisory board: Marius Wernig, a pioneer in cell reprogramming and Ramy Ibrahim, a leading innovator in the field of immuno-oncology. They join Chief Scientific Advisor Roger Pedersen, a central figure in the field of human stem cell biology.

Recent successes in cell therapy have sparked new hopes for the treatment of cancer. But the limited availability of human cells make them expensive and restrict their application. Drug development is also affected by the lack of human cells: differences between the animal models currently used and human biology are the major reasons why drugs fail in clinical trials.

Founded by stem cell biologist and neurosurgeon Mark Kotter, bit bio is commercializing Opti-OX, a proprietary technology platform for the efficient and consistent reprogramming of human cells for use in research, drug discovery, and cell therapy. With the expansion of its team, bit bio is entering its next phase of development to transform drug discovery and enable a new generation of cell therapies.

"The next generation of medicine hinges on widespread access to human cells," said Kotter. "That is the challenge we have set out to solve at bit bio, and I am incredibly proud to announce our founding team, which features innovators who have not only helped invent our field but who continue to push the envelope."

"At bit bio, we all share the same core ethos: democratizing access to human cells for the benefit of patients," added Chief Business Officer Paul Morrill. "I'm excited to join this world-class team as we work toward this ambitious goal."

Paul Morrill(PhD) joins the company as Chief Business Officer. Paul is an entrepreneur and scientist with over thirty years of experience in the biotech and pharma industries. Most recently, he was the commercial founder and President of Horizon Discovery Group, and founder of CellRx Limited, a growth factors company serving the biopharmaceutical, stem cell and research sectors. Paul holds a PhD in Biotechnology from the University of Cambridge.

Florian Schusterjoins as CFO / COO. Florian is an active entrepreneur and investor. Previously, he was CFO & Head of Strategic Partnerships for Tessa Therapeutics, a clinical-stage cell therapy company. Florian is a former investment banker, a graduate of the Stanford School of Engineering, and an alumnus of Harvard Business School.

Ramy Ibrahim(MD) joins as a member of the scientific advisory board. Ramy is a leading immuno-oncology clinician who has helped to develop some of the breakthrough therapies in this field. Ramy is currently the Chief Medical Officer at the Parker Institute of Cancer Immunotherapy. He has served as the Vice President of Clinical Development for Immuno-oncology at AstraZeneca, and as a member of the Bristol-Myers Squibb Immuno-oncology program.

Marius Wernig(MD PhD) joins as scientific advisory board member. Marius is a pioneer in cellular reprogramming. Marius' seminal 2010 paper in Nature demonstrating direct conversion of fibroblasts into neurons has sparked a widespread interest in cell reprogramming. His lab uses cellular reprogramming to understand how neurons are induced, and how they mature and maintain their identity. Marius is professor and co-director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine.

This team joins Mark Kotter and Roger Pedersen:

Mark Kotter(MD PhD, Founder/CEO)is a stem cell biologist and neurosurgeon at the University of Cambridge. By combining synthetic and stem cell biology, his team has developed a benchmark technology for the efficient and consistent production of human cells for use in research, drug development, and cell therapy. He is the founder of bit bio and co-founder of the cultured meat startup Meatable.

Roger Pedersen(PhD, Chief Scientific Advisor)is a pioneer and thought leader in the field of human stem cell biology. His lab was the first to isolate pluripotent epiblast stem cells from the epiblast layer of the developing mammalian embryo. In addition, he developed one of the first cellular reprogramming protocols. Roger was co-founder of the Cambridge Stem Cell Institute. His lab is currently focused on the differentiation of pluripotent stem cells, with potential applications for drug discovery, toxicity testing and cell therapies.

About bit biobit bio is the cell coding company. Based in Cambridge, UK,bit bio's team includes world leaders in stem cell biology, cellular reprogramming and cell therapy who are harnessing the power of synthetic biology to tackle the problem of inconsistency in the production of human cells. bit bio is developing Opti-OX, a proprietary technology platform capable of producing any human cell for research, drug discovery and cell therapy. For more information, visit bit.bio.

Press contact: Ben Kellogg, ben@bwkny.com +1 (917) 816-0831

SOURCE bit bio

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Pioneers of cellular reprogramming and cell therapies join bit bio - PRNewswire

DiscGenics Announces US Clinical Study of Cell Therapy for Disc Degeneration Clears Final Planned Safety Review – PRNewswire

The DSMC completed a third and final planned mid-trial safety review following treatment of the first six subjects in the high dose study cohort, each of whom was treated based on random assignment into one of three arms: high dose IDCT study treatment, vehicle or placebo.

The DSMC reported there were no safety issues and recommended that the study proceed with completion of patient enrollment with no changes to the protocol.

This news closely follows a recent company announcement that the second planned safety review of the first 30-subject low dose study cohort revealed no safety issues.

"We are delighted to commence the final enrollment stage in our U.S. study of IDCT for DDD and are thrilled that the first 36 patients have now been safely treated," said Flagg Flanagan, Chief Executive Officer and Chairman of the Board of Directors for DiscGenics. "This is a blinded, first-in-human study where neither the treating clinicians nor the patients know what treatment is being administered. As a result, performance of periodic safety checks by an unblinded and independent body was essential to ensuring the ongoing safety of IDCT in a clinical setting. The members of the DSMC played a critical role in this process and we would like to sincerely thank them for their time and thoughtful review of the blinded safety data."

Patient enrollment in this U.S. study will now continue through completion of 60 total subjects.

About the IDCT TrialThe IDCT trial is a prospective, randomized, double-blinded, vehicle- and placebo-controlled, multicenter clinical study to evaluate the safety and preliminary efficacy of IDCT in subjects with single-level, symptomatic lumbar intervertebral disc degeneration. The trial is underway in 14 centers across 12 states in the U.S. and will enroll 60 subjects. Those subjects who meet all eligibility criteria are being randomized to one of four treatment cohorts: low dose IDCT (n=20), high dose IDCT (n=20), vehicle (n=10) and placebo (n=10). Each subject receives a single intradiscal injection of his or her assigned treatment into the target symptomatic lumbar intervertebral disc. Following treatment, subjects will be observed and evaluated for a period of one year, with a one-year extension period. Primary outcome measures include safety and reduction in pain. Secondary outcome measures include reduction in disability and radiographic improvement.

Through this study, IDCT is being evaluated under an investigational new drug (IND) allowanceby the U.S. Food and Drug Administration (FDA) and will be regulated as a drug-biologic through a therapeutics biologics license application (BLA). Importantly, DiscGenics announced in August 2019that the FDA granted Fast Track designation for IDCT as a potential treatment option for chronic low back pain. For more information on the U.S. study, please visit: https://clinicaltrials.gov/ct2/show/NCT03347708.

IDCT is also being evaluated in a multicenter safety study in Japan, which is supported by a Clinical Trial Notification (CTN) approved by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). For more information on the Japanese study, please visit: https://clinicaltrials.gov/ct2/show/NCT03955315.

About DiscGenicsDiscGenics is a privately held, clinical stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. As the only company in the world to develop an allogeneic cell therapy derived from intervertebral disc cells to treat diseases of the disc, DiscGenics believes it has a unique opportunity to harness the restorative potential of the human body to heal millions of patients suffering from the debilitating effects of back pain. DiscGenics' first product candidate, IDCT, is a homologous, allogeneic, injectable cell therapy that utilizes biomedically engineered progenitor cells derived from intervertebral disc tissue, known as Discogenic Cells, to offer a non-surgical, potentially regenerative solution for the treatment of patients with mild to moderate degenerative disc disease. For more information, visit discgenics.com.

Media ContactLindsey Saxonlindsey@discgenics.com

SOURCE DiscGenics, Inc.

http://www.discgenics.com

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DiscGenics Announces US Clinical Study of Cell Therapy for Disc Degeneration Clears Final Planned Safety Review - PRNewswire

Medicare Coverage of CAR-T Cell Therapy Raises New Questions – The Heartland Institute

Still to be determined is how hospitals and other health care facilities will be reimbursed for the therapy and whether patients will have access to the therapy under health care proposals such as Medicare for All or a so-called public option.

One of the most promising cancer treatments to come along in years, CAR-T cell therapy uses the bodys own immune system to attack and kill cancer cells. The treatment involves bioengineering T cells, a white blood cell that fights foreign substances in the body, and equipping them with new Chimeric Antigen Receptors that target cancer cells.

CAR-T cell therapy has been approved by the U.S. Food and Drug Administration (FDA) for children with leukemia and adults with advanced lymphoma. The therapy is typically used alongside other, more traditional treatments such as surgery, chemotherapy, and radiation. Its use in combatting other forms of cancer is pending with the FDA, an agency known for its slow approval process.

Hefty Price Tag

There are currently two approved CAR-T treatments: Novartis Kymriah (tisagenlecleucel) and Gileads Yescarta (axicabtagene).

Like most newly introduced cutting-edge treatments, the two products come with a hefty price tag. A course of treatment of Kymriah costs $475,000 for pediatric and young adult patients with leukemia, and both are priced at $373,000 to treat lymphoma in adults, according tobiopharma.com. Under Centers for Medicare & Medicaid Services (CMS) regulations published in August, Medicare will reimburse hospitals for 65 percent of the treatments cost, or about $242,000, through Part B.

Although hospitals will likely welcome Medicares financial commitment, there still remains a sizable gap. Further complicating reimbursement is the lack of a separate Medicare billing code for CAR-T treatment, which will be handled via codes for bone marrow and stem cell transplants until a CAR-T billing code is developed, which could take up to three years.

CMS worked closely with the FDA and the National Cancer Institute in developing the new regulations, a time-consuming process rooted in the complexities of developing a reimbursement scheme and overseeing an innovative and evolving therapy.

At a July 31 Heritage Foundation panel discussion on Medicare for All, CMS Administrator Seema Verma discussed the challenges government programs face in approving innovative treatments.

Much of the problem is when Congress says you can cover durable medical treatment, supplies, and drugs, said Verma. Sounded great when they wrote that law 30 to 40 years ago but doesnt make sense in todays environment. All of these new treatments are coming out, and they dont fit nicely into the way the law has been constructed, and it creates problems for the agency.

Bonner R. Cohen,Ph.D.,(bcohen@nationalcenter.org)is a senior fellow at the National Center for Public Policy Research and a senior policy analyst with the Committee for a Constructive Tomorrow (CFACT).

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Medicare Coverage of CAR-T Cell Therapy Raises New Questions - The Heartland Institute

News – Michigan’s CON Board Sidelines Promising Cancer Treatment – The Heartland Institute

The commission adopted the rule at the behest of several of Michigans established cancer treatment providers and on the advice of a panel of clinical experts it appointed to evaluate the need for the regulation.

The panel also recommended requiring providers to be accredited by the Foundation for the Accreditation of Cellular Therapy (FACT), a national nonprofit organization that inspects cellular therapy facilities.

Keeping Out Competition

CON laws, which in some states are called Certificate of Public Need laws, are designed to keep health care providers from engaging in unnecessary capital outlays that would ultimately be passed on to patients in the form of higher costs. In practice, this means hospitals and other health care providers must get the approval of a state agency before offering new services, expanding their operations, or implementing new medical technologies. Dominant providers frequently use CON laws to limit competition from smaller hospitals.

CAR-T therapy is different from most health care services, states Anna Parsons, a policy coordinator with the American Legislative Exchange Council, because it does not require a capital investment (see commentary).

An FDA-certified hospital should be capable of offering these treatments, since all the high-tech bioengineering is done at other locations, Parsons told Reason.com.

Michigan As Outlier

Michigan state Sen. Curt VanderWall (R-Ludington), who chairs the Senate Health Policy and Human Services Committee, says he opposes CON regulation of CAR-T.

It is concerning to me that the CON commission expanded a requirement into a new clinical service area that goes well beyond the federal requirement, VanderWall toldHealth Care News.

The Centers for Medicare and Medicaid Services makes no mention of FACT accreditation in its August 7 press release stating Medicare will cover the proceedurein healthcare facilities enrolled in the FDA risk evaluation and mitigation strategies for FDA-approved indications.

The new CON requirement to obtain third-party accreditation will be a barrier to access, create an unnecessary financial burden for providers, and limit the sites of care from offering cellular therapies to patients, said VanderWall.

VanderWall says patients should be allowed to choose their own CAR-T provider.

Patient choice and access are priorities for me, said VanderWall. Patients will be able to work with their doctors to find the best treatment site based on safety and access.

Michigan should not be an outlier, VanderWall said. No other state has a CON standard for immune effector cell therapy (IECT), and safe treatment will be offered according to the established federal guidelines in the other 49 states.

A Joint Legislative Committee and the governor were allowed 45 days from the September 19 decision to review the new language to regulate IECT, of which CAR-T is a form, and the FACT accreditation requirement.

AnneMarie Schieber(amschieber@heartland.org)is managing editor ofHealth Care News.

Official Connections:

Michigan State Senator Curt VanderWall, (R Ludington):https://www.senatorcurtvanderwall.com

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News - Michigan's CON Board Sidelines Promising Cancer Treatment - The Heartland Institute

Thought Leadership & Innovation Foundation to Expand Its Regenerative Medicine Program Through New Collaboration with RenovaCare – Business Wire

MCLEAN, Va.--(BUSINESS WIRE)--The Thought Leadership & Innovation Foundation (TLI) announces today plans to build on its existing Regenerative Medicine Program through a research collaboration with cellular therapy industry leader RenovaCare. As part of TLIs efforts to conduct vital research in regenerative medicine and chronic disease, this initiative aims to innovate methods for reducing complications from burn and diabetic wounds across large populations.

Our research base, collaborative institutions and long history of innovation align with RenovaCares commitment to breakthrough biomedical technologies, says Bill Oldham, founder and chairman of the Board, TLI. The patented RenovaCare SkinGun technology and its ability to ultra-gently spray stem cells could present a special opportunity for investigations and applications in a wide range of regenerative therapies. Working together, our overall goal is to improve the quality, efficiency and effectiveness of patient care by not only developing new treatment methods, but also by making thoughtful and systematic changes to healthcare and health systems.

TLIs Regenerative Medicine program seeks to adapt new strategies based upon sound scientific evidence, utilizing its infrastructure to support the continuation of scientific and medical work, as well as the development of grant-funded research and other initiatives.

Dr. Robin A. Robinson, who is a TLI Fellow, Vice President of Scientific Affairs, RenovaCare, and named one of the top 100 innovators in medicine by Medicine Maker in 2018, states, This exciting collaboration between RenovaCare and TLIs Regenerative Medicine Program is the first step toward the development of meaningful and quality therapeutic treatments that will benefit patients around the world.

About TLI Foundation:

TLI Foundation is a nonprofit foundation focused on driving innovative thinking and action on global issues relating to health, education and economic empowerment. The organization is committed to fostering transformative change and improving the health and well-being outcomes of communities around the world. Visit https://www.thoughtfoundation.org/

About RenovaCare:

RenovaCare, Inc. is a biotechnology company focused on developing first-of-their-kind autologous (self-donated) stem cell therapies for the regeneration of human organs. Initial products under development target the bodys largest organ, the skin. Investigative clinical use of their flagship technology has shown to be promising new alternatives for patients suffering from burns, and chronic and acute wounds. https://www.renovacareinc.com.

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Thought Leadership & Innovation Foundation to Expand Its Regenerative Medicine Program Through New Collaboration with RenovaCare - Business Wire

Gene Therapy/Editing Series 1: A Brief Introduction To Gene Therapy – Seeking Alpha

The recent approval of various gene therapies, for example, Luxturna and Zolgesma and high premium acquisitions of gene therapy companies have shifted the investor focus to this rapidly growing biotechnology field. In this series of review articles, I will review the gene therapy and gene editing field, starting first with the basics, including a brief overview of the history of the field and then moving on to some technical aspects, for example, the manufacturing, different methods of delivery, and then moving on to discussing the competitive landscape covering one genetic disease in each article.

Let's first define what is a gene? A gene is a sequence of nucleotides in DNA that encodes the synthesis of a gene product, which is usually a protein.

(F8 gene, mutations in the gene cause Hemophilia A)

Usually, the code (in the form of a specific arrangement of nucleotides or base pairs contained in the gene is used to form mRNA (called transcription) which acts as a messenger to take the code to the target organ of the body. The information stored in the mRNA is then used to encode and synthesize the target protein (called translation) which then performs its intended function in the body.

(Steps involved in synthesizing a protein from the code in a gene)

An estimated number of protein-coding genes in the human body is approximately 20,000 to 25,000, which has been revised down from the initial prediction of 100,000 genes. Each gene contains a number of base pairs, the number of which is estimated to range from about 50 million to 300 million in the human body. In general, a gene therapy can be broadly defined as delivering in a new gene into the cells to compensate for a defective gene. In gene therapy, a newly delivered gene can perform different functions; for example, it can either replace the defective gene or it can silence an abnormal gene.

(An example of a gene therapy using an adenoviral vector to deliver the normal gene)

While I will discuss the various steps and delivery systems in gene therapy in detail later, viruses like lentiviruses and adenovirus are most commonly used as vectors in gene therapy. It was as early as 1950s that scientists first discovered that a virus can be used to inject the DNA in the cells of the host. In 1970s, various experiments started to use viruses as delivery systems for genes in the human body. In 1971, Merril, et al conducted a scientific experiment showing that DNA could be injected into the human cells to fix a biological problem in the cells. This group of scientists extracted the cells from patients suffering from a disease called Galactosemia. It is important to note that this first gene therapy experiment involves the manipulation of genes ex vivo, that is in cells growing in a petri dish outside the body in a lab, which is easier to perform than manipulating the genes inside the human body, called in vivo approach. In 1972, a famous article in the prestigious journal Science by authors Friedman and Roblin first proposed that the gene therapy may ameliorate some human genetic diseases in the future. During the 1980s, various scientists like Martin Cline and French Anderson conducted experiments on using viruses as delivery vehicles for DNA in human or mouse cells. The first human trials of gene therapy started in the late 1980s and the results were reported in early 1990s. One of the first reported clinical studies in humans involved ex vivo modification of white blood cells taken from patients with advanced melanoma, using a retroviral vector to insert a gene coding for interleurkin-2 and injecting the genetically altered cells back into the patients. During the 1990s, French Anderson reported a successful clinical trial where a retroviral vector was used to transfer a gene encoding for adenosine deaminase, ADA in children with severe combined immunodeficiency, SCID. During the 1990s, most of the work in gene therapy continued in the therapeutic area of ADA-SCID.

Despite reasonably successful clinical results, the field of gene therapy suffered a serious setback in 1999. Jesse Gelsinger, an 18-year-old patient with a disease called ornithine transcarbamylase, OTC deficiency, which results due to a missing gene coding OTC died 4 days after receiving the gene therapy in a clinical trial conducted by the University of Pennsylvania due to massive immune response resulting in multi-organ failure. As a result, FDA put a suspension on various gene therapy clinical trials.

The field of gene therapy was then suspended for almost a decade. Glybera, a gene therapy was approved in Europe for reading a genetic disease, lipoprotein lipase deficiency in 2012. However, Glybera was a commercial failure after insurers in Europe were reluctant to pay for its expensive $1 million per patient tag. Finally, uniQure (QURE) the company that developed Glybera discontinued it.

Another commercial gene therapy failure was Strimvelis, a stem cell gene therapy to treat ADA-SCID. Despite its price being lower than Glybera ($665,000 per year), the therapy was not commercially successful in Europe and was sold by GlaxoSmithKline (GSK) to Orchard Therapeutics (ORTX) in 2018. In the US, the first approved gene therapy was Kymriah, an autologous CAR-T therapy to treat autologous lymphoblastic leukemia (ALL), which was developed by Novartis (NYSE:NVS).

After Kymriah, another autologous CAR-T therapy, Yeskarta (by Kite Pharmaceuticals) was approved by FDA to treat adult diffuse large B-cell lymphoma. Kite was later acquired by Gilead (NASDAQ:GILD). The first in vivo gene therapy approval in the US was Luxturna, an AAV gene therapy for patients with RPE 65 mutation-associated retinal dystrophy, which was developed by Spark Therapeutics which also was later acquired. Luxturna was another major milestone in the history of gene therapy as it resulted in a miraculous effect of restoring vision to children who were blind since birth. Recently, bluebird bio's (BLUE) gene therapy for transfusion-dependent beta-thalassemia was approved in Europe.

The developmental landscape of gene therapies can be summarized in some excellent figures from the journal Molecular Therapy published by the American Society of Gene and Cell Therapy (ASGCT). A group of researchers reviewed the medical literature and identified 336 gene therapies being developed for 138 different clinical indications covering 165 genetic targets excluding oncology. The researchers found that 74% of these 336 gene therapies were concentrated in five medical specialties, that is, hematology, endocrinology, neurosciences, cardiology, and ophthalmology. When classifying by different disease families, inborn errors of metabolism was the disease category with a majority of ongoing gene therapy trials.

(Landscape of gene therapy programs by organ system and disease area, source: Mol. Therapy)

When looking at specific clinical indications, Duchenne muscular dystrophy (DMD) was the clinical indication with the highest number of gene therapies being developed (15). HIV gene therapies (12 gene therapy programs) and hemophilia (11 gene therapy programs) took the second and third place respectively.

In terms of the number of gene therapy/editing programs being developed by a particular company or organization, Sangamo Therapeutics (SGMO) took the top spot (see the figure below).

(Landscape of gene therapy programs by company/organization, source: Mol. Therapy)

In conclusion, gene therapy has recovered from its earlier setbacks to emerge as one of the most innovative areas in biotechnology. In this first article of the series, I have provided a brief background about gene therapy, its history, and a broad top-down landscape. In the next article in the premium service, I will discuss various delivery systems for gene therapy.

A free two-week trial for the premium Marketplace service is open for another week only. Only 25 more spots left.

Disclosure: I am/we are long BLUE, ORTX, QURE, SGMO, AXGT, CRSP. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Gene Therapy/Editing Series 1: A Brief Introduction To Gene Therapy - Seeking Alpha

Discovery in Monkeys Could Lead to Treatment for Blindness-Causing Syndrome – Technology Networks

A genetic mutation that leads to a rare, but devastating blindness-causing syndrome has been discovered in monkeys for the first time. The finding offers a promising way to develop gene and cell therapies that could treat the condition in people.

Three rhesus macaques with a mutated gene that's associated with Bardet-Biedl Syndrome have been discovered, according to a study published in the journal Experimental Eye Research. It is the first known naturally occurring nonhuman primate model of the syndrome, which is also called BBS.

BBS leads to vision loss, kidney dysfunction, extra fingers or toes, and other symptoms. It occurs in 1 of 140,000 to 160,000 North American births.

"There is no cure for Bardet-Biedel Syndrome today, but having a naturally occurring animal model for the condition could help us find one in the future," said the paper's corresponding author, Martha Neuringer, Ph.D., a professor of neuroscience at the Oregon National Primate Research Center at Oregon Health & Science University, and a research associate professor of ophthalmology in the OHSU School of Medicine and OHSU Casey Eye Institute.

Broader applications

Rhesus macaques with this disease could help more than just BBS patients. BBS is part of a larger family of diseases called retinitis pigmentosa, all of which affect the retina, or the back part of the eye. A naturally occurring animal model for BBS could help researchers find treatments for a variety of retinitis pigmentosa diseases.

The discovery is timely, as gene therapy is already becoming a reality for some with retinal diseases. In the 1990s, researchers discovered dogs that had a gene mutation linked to a congenital blindness-causing condition called Leber's congenital amaurosis. That animal model played a key role in helping researchers develop what became the first FDA-approved gene therapy for an inherited disease in December 2018. Neuringer's group also hopes to develop a similar therapy for BBS.

Gene mutation

After Neuringer and colleagues discovered two related monkeys without cells that are key to vision, OHSU nonhuman primate genetics experts Betsy Ferguson, Ph.D., and Samuel Peterson, Ph.D., examined the animals' genomes. They quickly found both monkeys had a mutation of the BBS7 gene, one of at least 14 genes associated with BBS.

Because Ferguson leads an effort to genetically sequence 2,000 rhesus macaques at the nonhuman primate research center, they were also able to search the genomes of numerous other monkeys there. As a result, the team found a third monkey with the same mutation. The third rhesus macaque already had serious vision loss when it was identified in 2018 at age three and a half, although it adapted so well among its social group that the vision loss wasn't obvious. Neuringer's team is observing the third monkey over time to better understand how the disease progresses in rhesus macaques.

Neuringer and her colleagues are now using a National Eye Institute grant to breed more animals with the naturally occurring BBS7 mutation. Having more animals with the mutation can help researchers better understand the disease and test potential treatments. The knowledge they gain could enable them to develop gene and cell therapies that could cure BBS and related retinal degenerative diseases.

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

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Discovery in Monkeys Could Lead to Treatment for Blindness-Causing Syndrome - Technology Networks

Animal Stem Cell Therapy Market Revenue, Opportunity, Segment and Key Trends 2017 2025 – Health News Office

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Animal Stem Cell Therapy Market Revenue, Opportunity, Segment and Key Trends 2017 2025 - Health News Office

Intellia Therapeutics Presents In Vivo and Ex Vivo Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) -…

CAMBRIDGE, Mass., Oct. 24, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology is presenting one oral presentation and four poster presentations at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) meeting taking place October 22-25, 2019, in Barcelona, Spain.

We are excited to share progress across Intellias in vivo and ex vivo programs at this important scientific venue, said Laura Sepp-Lorenzino, Ph.D., chief scientific officer, Intellia Therapeutics. Our data shows the complexity of the edits we are able to make with CRISPR/Cas9, while achieving important therapeutically relevant results. We are building on the success of our modular platform now having demonstrated consecutive targeted knockout and insertion genome edits in preclinical studies. Additionally, we presented data from our engineered cell therapy program, which continues to demonstrate the use of CRISPR/Cas9 for combined knockout and targeted integration in human T cells.

Intellia Demonstrates Consecutive In Vivo Genome Editing in Alpha-1 Antitrypsin Deficiency Mouse Model

Intellias oral presentation highlights its alpha-1 antitrypsin deficiency (AATD) study showing that consecutive dosing of two distinct lipid nanoparticle (LNP) formulations, in adultmice, achieves two targeted genome editing events, resulting in knocking out the faulty gene and restoring therapeutic levels of normal alpha-1 antitrypsin protein (hAAT). Intellias approach for AATD uses a modular hybrid delivery system combining a non-viral LNP which encapsulates CRISPR/Cas9 with an adeno-associated virus (AAV) carrying donor DNA template. Compared to traditional viral-based delivery of gene editing components, Intellias LNP delivery system can overcome the inherent limitations of immunogenicity to facilitate multiple in vivo gene editing events.

In a mouse model harboring the human PiZ allele, the most severe genetic defect in AATD patients, Intellia first reduced expression of the defective protein using gene knockout. Three weeks following the PiZ allele knockout, Intellia inserted the normal human alpha-1 antitrypsin gene, resulting in stable (throughout 12 weeks of observation), therapeutically relevant circulating protein levels. In the study, a sustained reduction of the circulating PiZ protein levels of >98% was observed for over 15 weeks. This is the first in vivo demonstration of a non-viral delivery platform, enabling a consecutive dosing approach for achieving multiple genome edits in the same tissue of the same animal. Intellias oral presentation, titled In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression, will be given by Anthony Forget, Ph.D., on October 25, 2019. This presentationwill be available on Intellias website at http://www.intelliatx.com.

Intellias Poster Presentations

WT1-Specific TCR Engineered Cell Therapy Studies

Intellia presented new in vitro data showing that CRISPR/Cas9-mediated genome editing for in locus insertion, combined with endogenous T Cell Receptor (TCR) knockout, leads to significant reduction in mispairing of endogenous and transferred TCR chains. This approach is expected to generate transgenic-TCR (tg-TCR) T cell therapies for hematological cancers and solid tumors. Results demonstrate a highly efficient reduction of >98% in endogenous TCR and chains while reaching >70% insertion rates of tg-TCRs without further purification. The poster titled Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In Locus Insertion Combined with Endogenous TCR Knockout, was presented on October 24, 2019, by Birgit Schultes, Ph.D.

Researchers also presented in vitro data showing that a library of WT1-specific TCRs were generated, several of which Intellia is currently evaluating as part of its lead engineered cell therapy program targeting Acute Myeloid Leukemia (AML). This presentation, Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia, was presented on October 23, 2019 by Intellias collaborator, Erica Carnevale, Ph.D., IRCCS Ospedale San Raffaele.

Primary Hyperoxaluria Study

Intellia showed the continued progression of its modular platform capability using CRISPR/Cas9 to knockout either hydroxyacid oxidase 1 (Hao1) or lactate dehydrogenase A (Ldha), leading to a dose-dependent and persistent reduction of urinary oxalate levels in a Primary Hyperoxaluria Type 1 (PH1) mouse model. Data shows Ldha gene disruption also decreased LDH enzyme activity in the liver and did not impair the disposition of lactate in either wild type or renally-impaired mice. These results highlight the potential of editing genes in the glyoxylate detoxification pathway using a non-viral delivery approach as a one-time treatment option for PH1. These data were presented as a poster, titled CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria, by Sean Burns, M.D., on October 24, 2019.

Off-Target Screening Platform

Intellia demonstrated its approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Results from targeted off-target sequencing in edited cells showed that biochemical off-target discovery approaches were the most sensitive and accurate. These data were presented as a poster on October 23, 2019, titled In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics, by Dan OConnell, Ph.D.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms such as its AATD program; develop our proprietary LNP-AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half of 2021.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position, including through our arbitration proceedings against Caribou; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations withNovartisor Regeneron or its otherex vivocollaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, andIntellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Presents In Vivo and Ex Vivo Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) -...

Researchers Used Green Tea as a ‘Remote Control’ to Activate Cell Therapies for Diabetes – D-brief – Discover Magazine

(Credit: Kirasolly/Shutterstock)

Since ancient times,the health benefits of green tea have been the stuff of legend. Now,researchers are turning to the antioxidant-rich leaves for a decidedly modern purpose triggering cell-based therapies.

In a study published Wednesday in Science Translational Medicine, a team of researchers with East China Normal University and First Affiliated Hospital of Shenzhen University found that green tea can work to activate lab-made cells for disease treatment. In short, they used the beverage as a drinkable remote control.

And the authors think that the new approach, which they used as part of a successful treatment for diabetes in mice and monkeys, could one day be used to deliver remote controlled, easy-to-follow therapies for diabetes in humans.

In recent years, scientists have worked to refine how cell therapies which have shown promise in treating diseases like cancer are controlled. These therapies often rely on a chemical compound to trigger cells to secrete a specific therapeutic substance. But many of the currently-used triggers, like antibiotics, can have negative side effects or are unsafe for prolonged use.

In search of a bettertrigger, lead author Jianli Yin and her colleagues looked for a compound thatwas natural, nontoxic and perhaps even beneficial to health. Enter green tea.

In addition to itslaundry list of purported health benefits, green tea contains a molecule calledprotocatechuic acid, or PCA. The research team engineered gene switches thatcould be activated by PCA before inserting them in different human and animalcells.

To test out theirnew system, the team implanted the PCA-responsive cells into mice with diabetes,a disease that impairs the bodys ability to produce insulin. After the micewere given concentrated doses of green tea, the researchers observed that therodents insulin levels rose while their blood sugar levels fell.

But the studyauthors also noted that what works in mice doesnt always translate to humans.So, to see if they achieved the same results in our nonhuman primate cousins, thescientists tested their system on macaque monkeys, too.

The findings were indeedsimilar after drinking green tea or receiving an injection of PCA, the monkeysinsulin production returned to normal.

And while there havent yet been clinical studies on similar cell-based therapies in humans, the researchers remain optimistic that their findings could present new possibilities for disease treatment. That is, just so long as you dont mind drinking a little green tea.

Green tea has been an extremely popular beverage for more than 2,000 years, the study said. We suspect that [this system] will be considered safe and help to improve and encourage patient compliance.

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Researchers Used Green Tea as a 'Remote Control' to Activate Cell Therapies for Diabetes - D-brief - Discover Magazine