Daily Archives: October 15, 2019

ZUG, Switzerland and CAMBRIDGE, Mass., Oct. 15, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and KSQ Therapeutics, a biotechnology company using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development, today announced a license agreement whereby CRISPR Therapeutics will gain access to KSQ intellectual property (IP) for editing certain novel gene targets in its allogeneic oncology cell therapy programs, and KSQ will gain access to CRISPR Therapeutics IP for editing novel gene targets identified by KSQ as part of its current and future eTIL (engineered tumor infiltrating lymphocyte) cell programs. The financial terms of the agreement are not being disclosed.

We are thrilled to gain access to CRISPR Therapeutics foundational IP estate through this agreement, said David Meeker, M.D., Chief Executive Officer at KSQ Therapeutics. Our eTIL programs involve editing gene targets in human TILs that were discovered at KSQ by applying our proprietary CRISPRomics approach to immune cells in multiple in vivo models. This agreement clears an important path for us to be able to bring these programs through development and commercialization, leveraging CRISPR Therapeutics proprietary editing technology.

The gene targets within the scope of the license agreement were identified using KSQs proprietary CRISPRomics drug discovery engine, which allows genome-scale, in vivo validated, unbiased drug discovery. These specific targets were uncovered in screens to identify genetic edits that could enhance the functionality and quality of adoptive cell therapies in oncology.

KSQ has built an industry-leading platform to screen for novel gene targets using its technology, and has identified a group of targets that could help unlock the full potential of adoptive cell therapy in oncology, said Samarth Kulkarni, Ph.D., Chief Executive Officer at CRISPR Therapeutics. As a result of this license agreement, CRISPR Therapeutics will have the opportunity to bring these novel targets into our leading allogeneic CAR-T development platform to further strengthen our future programs in this important therapeutic area.

About KSQ TherapeuticsKSQ Therapeutics is using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development. The company is advancing a pipeline of tumor- and immune-focused drug candidates for the treatment of cancer, across multiple drug modalities including targeted therapies, adoptive cell therapies and immuno-therapies. KSQs proprietary CRISPRomics drug discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. KSQ was founded by thought leaders in the field of functional genomics and pioneers of CRISPR screening technologies, and the company is located in Cambridge, Massachusetts. For more information, please visit the companys website at http://www.ksqtx.com.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR Therapeutics Investor Contact:Susan Kim+1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Jennifer PaganelliWCG on behalf of CRISPR+1 347-658-8290jpaganelli@wcgworld.com

KSQ Contact:Michael LampeTel: 484-575-5040michael@scientpr.com

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CRISPR Therapeutics and KSQ Therapeutics Announce License Agreement to Advance Companies' Respective Cell Therapy Programs in Oncology - BioSpace

A phase I/II trial run by the Dutch company ProQR has found that its RNA therapy could significantly improve the vision of people with Lebers congenital amaurosis, a rare genetic disease for which there is no treatment.

The RNA drug, called sepofarsen, is designed to treat people with a specific mutation in a gene called CEP290. This mutation causes the RNA transcript of the gene to have the wrong three-dimensional structure, blocking its translation into a protein. This, in turn, causes vision loss in the first few years of life.

Sepofarsen is an RNA molecule that specifically binds to the faulty RNA transcript to stabilize its structure and allow the retinal cells to produce the protein.

In a phase I/II trial run in the US and Belgium, the RNA drug significantly improved the vision of children and adults with this condition over a 1-year period.

In some cases the patients vision improved to a level that could be deemed life-changing, said Stephen Russell, a professor at the University of Iowa and principal investigator of the study.

The effects of the drug were stronger on patients that had a certain level of visual acuity to start with. These are ultimately the target population of ProQR, which is already running a phase II/III study that will follow the response of 30 patients over the course of 2 years. Results from that trial are expected in 2021 and will inform whether the FDA and the EMA approve the drug or not.

The main goal of the phase I/II trial was to determine the safety of sepofarsen. While the treatment caused cataracts in eight out of 11 patients, all of those who underwent lens replacement surgery recovered their vision. Other side effects of the drug on the eye were manageable with additional treatments.

There are hundreds of different genetic mutations that cause blindness. The rarity of each of these conditions individually has meant that many of them have no treatment available. In recent years, gene therapy has become an option to treat some of these conditions; the first was Luxturna, approved in 2017. Another approach that has only entered the first clinical trial this year is CRISPR gene editing, which is being carried out by Editas Medicine and Allergan.

In contrast, ProQRs RNA drug could provide an alternative approach that does not involve a permanent change in the DNA of retinal cells. The drug is instead delivered to the eye via injection every 6 months.

Still, each of these new treatments can only address one specific mutation of the many causing blindness. As all these new technologies are developed, together they could eventually provide solutions covering a wide range of these mutations.

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RNA Therapy Improves Vision in Untreatable Genetic... - Labiotech.eu

Burn specialists at Massachusetts General Hospital are the first in the world to successfully use live-cell, genetically engineered pig skin to temporarily close a burn wound in a human patient but the breakthrough has drawn opposition from People for the Ethical Treatment of Animals.

The ultimate holy grail is the end to the worlds organ shortage, that would be the holy grail, this has come at a time when genetic editing is really hot and what we could do in years, we can do in weeks, said Dr. Jeremy Goverman of the MGH Sumner Redstone Burn Service.

The pig tissue, known as xenoskin, was transplanted directly onto a human burn wound next to a larger piece of human skin.

Five days later, surgeons removed the human skin and the pig tissue to see that both grafts were stuck to the wound bed and were indistinguishable from each other.

Following the procedure, the burn wound was then treated further with a skin graft taken from the patients thigh. Healing progressed well and the patient will return to work soon.

The goal is to replace skin with xenoskin thats like it enough that it doesnt get rejected, said Goverman. Down the line we hope to ultimately create something thats not temporary.

The biggest push now is actually decreasing your donor site size and decreasing how much skin you have to harvest, said Goverman.

Patients who receive this type of graft typically have severe burns that require more than one operation and about a week of hospitalization.

Weve been using dressing like this in the past, we just havent been able to use anything with live cells. The live cells have all the appropriate factors that could really stimulate and regenerate and close our wounds for us, said Goverman.

MGH worked with Boston-based XenoTherapeutics, which designed the safety protocols for the special live-pig tissue graft.

Paul Holzer, CEO of XenoTherapeutics said, We have taken a small but unprecedented step in bringing xenotransplantation from theory to therapy, one that we hope will advance this promising field of medicine and benefit patients around the world.

Human skin grafts are subject to a national shortage and can be expensive, therefore using the pig skin can serve as a viable alternative, according to MGH.

But Alka Chandna, vice president of laboratory investigations cases at PETA, said there is no shortage of donated skin grafts.

Its categorically unethical to steal organs from another sentient being whos still using them. Pigs are individuals, not warehouses for spare parts, said Chandna.

Chandna said, Tinkering with the genes of these intelligent, sensitive beings to turn them into organ factories is a waste of lives, time and money and the suffering caused is unimaginable.

The advancement of the procedure reaches back decades to genetically modified pigs that were developed in the 1990s at MGH by Dr. David Sachs.

The modifications removed a gene specific to pigs and not present in humans, allowing the pig skin to appear less foreign to the human immune system.

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MGH doctors perform first-ever live-cell pig skin graft to burn patient - Boston Herald

The company expands its Scientific Advisory Board with four additional RNA splicing, genetics, and disease experts, who join SAB Chair Professor Tyler Jacks & special advisor Professor Phil Sharp as well as several other internationally-recognized SAB members.

WALTHAM, Mass., Oct. 15, 2019 /PRNewswire/ -- Skyhawk Therapeutics, Inc. ("Skyhawk"), a drug discovery and development company focused on revolutionizing disease treatment with small molecules that modify RNA expression, today announced the appointment of four additional internationally recognized experts in RNA biology and disease to its Scientific Advisory Board.

Skyhawk Therapeutics, Inc. (PRNewsfoto/Skyhawk Therapeutics)

"I am thrilled that we have assembled such a stellar group of RNA biology and human disease experts for Skyhawk's Scientific Advisory Board," said Prof. Tyler Jacks, Director of MIT's Koch Institute for Integrative Cancer Research and Chair of Skyhawk's SAB. "We look forward to having their combined knowledge and wisdom help guide Skyhawk's research and development efforts, to progress even more rapidly towards groundbreaking new approaches and therapies for patients with a variety of difficult-to-treat diseases."

Prof. Ben Blencowe is an internationally recognizedRNA biologist who has made pioneering contributions to the understanding of the molecular mechanisms controlling alternative splicing and their roles in evolution, development and disease. He holds the Banbury Chair of Medical Research and is Professor in the Donnelly Centre at the University of Toronto; he also serves as Director of the Donnelly Sequencing Centre. Prof. Blencowe has received numerous awards and honors for his research excellence and was recently elected Fellow of the Royal Society (UK).

Dr. Ben Ebert is the George P. Canellos, MD and Jean S. Canellos Professor of Medicine at Harvard Medical School, and Chair of Medical Oncology at the Dana-Farber Cancer Institute. His research focuses on the genetics, biology, and therapy of myeloid malignancies. His work has led to the characterization of clonal hematopoiesis as a pre-malignant state for hematologic malignancies, and elucidation of the mechanism of action of lenalidomide and related molecules that induce degradation of specific proteins. Dr. Ebert has served as president of the American Society for Clinical Investigation and is an elected member of the National Academy of Medicine and the Association of American Physicians.

Prof. Jeannie T. Lee is Professor of Genetics and Pathology at Harvard Medical School, the Blavatnik Institute, and the Massachusetts General Hospital. She specializes in the study of epigenetic regulation by long noncoding RNAs and uses X-chromosome inactivation as a model system. Prof. Lee also translates basic knowledge to find treatments for genetic disorders and co-founded two publicly traded companies Translate Bio and Fulcrum Therapeutics. She is a Member of the National Academy of Sciences, a 2018 Harrington Rare Disease Scholar, the 2016 recipient of the Lurie Prize, a 2016 recipient of the Centennial Award from the Genetics Society of America, the 2010 awardee of the Molecular Biology Prize from the National Academy of Sciences, and a Fellow of the American Association for the Advancement of Science.

Prof. Maurice Swanson is an expert on the regulation of RNA alternative processing during mammalian development and how this regulation is disrupted in neurological and neuromuscular diseases, including some types of muscular dystrophy and amyotrophic lateral sclerosis (ALS). Prof. Swanson is a Professor in the Department of Molecular Genetics and Microbiology at the University of Florida College of Medicine and Associate Director of the Center for NeuroGenetics. His lab focuses on the functions of repetitive DNA elements, particularly microsatellites or short tandem repeats (STRs), in RNA-mediated disorders. An important objective of these studies is to enhance tissue regeneration following treatment modalities designed to block the toxicity of STR.

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These four new members join Skyhawk's existing Scientific Advisory Board members & advisors including:

About Skyhawk TherapeuticsSkyhawk Therapeutics is committed to discovering, developing and commercializing therapies that use its novel SkySTAR (Skyhawk Small molecule Therapeutics for Alternative splicing of RNA) platform to build small molecule drugs that bring breakthrough treatments to patients.

For more information visit: http://www.skyhawktx.com, https://twitter.com/Skyhawk_Tx, https://www.linkedin.com/company/skyhawk-therapeutics/

SKYHAWK MEDIA CONTACT:Anne Deconinckanne@skyhawktx.com

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World Renowned Experts Appointed to Skyhawk Therapeutics Scientific Advisory Board - Yahoo Finance

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Mouse study shows immune cells drive brain damage

A neuron containing tangles of tau protein is surrounded by immune cells known as microglia in this computer-generated image. A study from Washington University School of Medicine in St. Louis has found that microglia drive neurodegeneration in diseases, including Alzheimer's disease, that are linked to tau protein. Targeting microglia may help treat such diseases.

Messy tangles of a protein called tau can be found in the brains of people with Alzheimers disease and some other neurodegenerative diseases. In Alzheimers, the tangles coalesce just before tissue damage becomes visible in brain scans and people start to become forgetful and confused.

Now, a new study has found that brain immune cells called microglia which are activated as tau tangles accumulate form the crucial link between protein clumping and brain damage. The research, published Oct. 10 inthe Journal of Experimental Medicine, shows that eliminating such cells sharply reduces tau-linked brain damage in the mice and suggests that suppressing such cells might prevent or delay the onset of dementia in people.

Right now many people are trying to develop new therapies for Alzheimers disease, because the ones we have are simply not effective, said senior authorDavid Holtzman, MD, the Andrew B. and Gretchen P. Jones Professor and head of theDepartment of Neurology. If we could find a drug that specifically deactivates the microglia just at the beginning of the neurodegeneration phase of the disease, it would absolutely be worth evaluating in people.

Under ordinary circumstances, tau contributes to the normal, healthy functioning of brain neurons. In some people, though, it collects into toxic tangles that are a hallmark of neurodegenerative diseases such as Alzheimers and chronic traumatic encephalopathy, a progressive brain disease often diagnosed in football players and boxers who have sustained repeated blows to the head. Holtzman and colleagues previously had shown that microglia limit the development of a harmful form of tau. But the researchers also suspected that microglial cells could be a double-edged sword. Later in the course of the disease, once the tau tangles have formed, the cells attempts to attack the tangles might harm nearby neurons and contribute to neurodegeneration.

To understand the role of microglial cells in tau-driven neurodegeneration, Holtzman, first author and postdoctoral researcher Yang Shi, PhD, and colleagues studied genetically modified mice that carry a mutant form of human tau that easily clumps together. Typically, such mice start developing tau tangles at around 6 months of age and exhibiting signs of neurological damage by 9 months.

Then, the researchers turned their attention to the gene APOE. Everyone carries some version of APOE, but people who carry the APOE4 variant have up to 12 times the risk of developing Alzheimers disease compared with those who carry lower-risk variants. The researchers genetically modified the mice to carry the human APOE4 variant or no APOE gene. Holtzman, Shi and colleagues previously had shown that APOE4 amplifies the toxic effects of tau on neurons.

For three months, starting when the mice were 6 months of age, the researchers fed some mice a compound to deplete microglia in their brains. Other mice were given a placebo for comparison.

The brains of mice with tau tangles and the high-risk genetic variant were severely shrunken and damaged by 9 months of age as long as microglia were also present. If microglia had been eliminated by the compound, the mices brains looked essentially normal and healthy with less evidence of harmful forms of tau despite the presence of the risky form of APOE.

Further, mice with microglia and mutant human tau but no APOE also had minimal brain damage and fewer signs of damaging tau tangles. Additional experiments showed that microglia need APOE to become activated. Microglia that have not been activated do not destroy brain tissue or promote the development of harmful forms of tau, the researchers said.

Microglia drive neurodegeneration, probably through inflammation-induced neuronal death, Shi said. But even if thats the case, if you dont have microglia, or you have microglia but they cant be activated, harmful forms of tau do not progress to an advanced stage, and you dont get neurological damage.

The findings indicate that microglia are the linchpin of the neurodegenerative process and an appealing target of efforts to prevent cognitive decline in Alzheimers disease, chronic traumatic encephalopathy and other neurodegenerative diseases. The compound Holtzman and Shi used in this study has side effects that make it a poor option for drug development, but it could point the way to other compounds more narrowly tailored to microglia.

If you could target microglia in some specific way and prevent them from causing damage, I think that would be a really important, strategic, novel way to develop a treatment, Holtzman said.

Shi Y, Manis M, Long J, Wang K, Sullivan PM, Remolina J, Hoyle R, Holtzman DM. Microglia drive APOE-dependent neurodegeneration in a tauopathy mouse model. Journal of Experimental Medicine.Oct. 10, 2019. DOI: 10.1084/jem.20190980

This study is supported by the National Institutes of Health (NIH), grant numbers NS090934 and AG047644; JPB Foundation; and the Cure Alzheimers Fund.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Targeting immune cells may be potential therapy for Alzheimer's - Washington University School of Medicine in St. Louis

For the last seven years, a rare neurological disorder ravaged Mitchell Herndon's body.

As the condition a genetic mutation diagnosed in only a few people in the world robbed the Missouri teen of his ability to walk, took his hearing and then his eyesight, Herndon made a decision: If the disease killed him, he would donate his body to science in the hopes of saving others.

Last Wednesday, just days before a potentially life-saving drug would have been made available to him, Herndon, 19, died. Abiding by his wishes, Herndon's family chose to gift his body to Washington University in St. Louis for research into neuro-muscular diseases something that doctors say will be invaluable for advancing the understanding of more than his own disorder.

"It's an incredible tool which he has donated. This will have an impact for many people that get identified with his condition in the future, as well as other people with other neurodegenerative conditions" such as amyotrophic lateral sclerosis (ALS), or possibly Alzheimer's and Parkinson's diseases, said Dr. Bob Bucelli, the neurologist who treated Herndon for the past year and an associate professor of neurology at Washington University School of Medicine in St. Louis. "It's a limitless resource that he's given and incredible what he's offered the medical community by doing that."

In May 2019, Herndon was the subject of an NBC News Digital documentary viewed 4 million times about what it was like to be living with a mysterious disease that kept progressing as doctors raced to try to save him.

Herndon, of Affton, Missouri, had been a healthy, athletic child when he started experiencing difficulty moving his legs at age 12. He was eventually diagnosed with a rare mutation of the ACOX1 gene, which until recently had only been diagnosed in one other person: a teenage girl in South Korea who is unable to communicate. Because the condition is so uncommon, it does not yet have a name.

Over the years, Herndon was in and out of the hospital. He lost the ability to walk multiple times, gaining it back to some degree thanks to physical therapy and medications until a relapse last fall left him in a wheelchair.

While many patients with extraordinarily rare diseases now find others like themselves thanks to genetic testing being cheaper and more widely available, Herndon never did. He found some companionship in the deaf community and among others with muscular disorders, but told NBC News in May that he would have loved to meet someone who could relate to the ups and downs of his particular disease.

If I knew someone who was 50 years old and had the same thing, if they were doing amazing, that would clear up a lot of anxiety."

If I knew someone who was 50 years old and had the same thing, if they were doing amazing, that would clear up a lot of anxiety, Herndon said at the time. If we found out this is progressive, that would suck, but at least I would know what to expect.

Despite the unique challenges Herndon faced, he kept a positive outlook often using humor to lighten the mood during his lengthy hospital stays, spending as much time as he could with his siblings, Maxwell, 17, and Miranda, 11, and when he was well enough attending St. Louis University where he enjoyed studying political science and theology.

Researchers knew Herndon's condition was going to get worse, but they were not sure how quickly he might decline. Then they stumbled upon something that they believed could stop the progression and possibly save his life.

Dr. Hugo Bellen, an investigator with the Howard Hughes Medical Institute and a professor at Baylor College of Medicine who studies genetics and neurobiology, was studying Herndon's mutation in fruit flies. Bellen discovered that a powerful antioxidant, NAC-Amide, showed promising results in stopping the disease's decline. But the medication was not approved by the Food and Drug Administration for use in patients yet.

Bucelli, Herndon's neurologist, worked tirelessly with the FDA to establish a protocol for the medication that would have been considered safe to try on Herndon. As Herndon worsened during his most recent hospital stay, eventually becoming unresponsive and going on life support, the FDA finally granted approval for Herndon to try it barely an hour after an MRI showed the disease had spread to his brain.

With irreversible brain damage, it was too late to try the drug. Herndons family made the painful decision to remove him from life support something Herndon had expressed to them that he wanted should he ever get to that point. The following day, held by his father, mother and brother, Herndon died, his mother, Michele Herndon, said.

While the drug approval came too late for Herndon, doctors have identified another patient who appears to have the same type of mutation as him: a young child in Ohio. Bucelli said he is sending the research he did on Herndon to the physicians in Ohio, which should open the door for them be able to receive the drug for their patient.

"Mitchell could potentially have a direct impact on this next patient."

"Mitchell could potentially have a direct impact on this next patient," Bucelli said.

In the meantime, Bellen has submitted the first-ever paper on Herndon's particular mutation for publication in a scientific journal. He said he suspects there are more patients who will be discovered to have the condition, and in the paper, Bellen proposed a name for it: Mitchell Disease.

Herndon's parents said their son was always eager to help medical professionals, whether it was letting students practice taking medical histories on him or allowing newer nurses to do procedures on him, even if there were more experienced nurses available. His mother said she hoped helping to find cures for this condition as well as others will be part of her son's legacy.

"Our decision to donate his body was just another way that we know he will continue to advance medical research and hopefully pave the way for future patients with his genetic mutation," Michele Herndon told NBC News via email. "We always knew that he believed his body was just that a body. And his soul is what would live on."

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This teen had a disease so rare, it didn't have a name. His legacy could help countless others - NBC News

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

One of those companies, Mogrify, just raised $16 million in Series A financing to bring its overall funding to over $20 million since its February launch. Led by CEO Darrin Disley, the funding will help expand their new base in Cambridge to a 60-strong staff and push forward their direct-conversion approach to cell therapy through research and licensing. Investors include Parkwalk Advisors and Ahren Innovation Capital.

They list potential applications as treatments for musculoskeletal and auto-immune disorders, cancer immunotherapy, and therapies for ocular and respiratory diseases. For example, you could use it regenerate cartilage in arthritis patients.

If you could take a cell from one part of the body and turn it into any other cell at any other stage of development for another part of the body, you effectively have the Holy Grail of regenerative medicine, Disley told Labiotech.eu in April.

Mogrifys advantage over the Yamanaka method called induced pluripotent stem cells (iPS), is that in theory it can be more scalable and avoid the problems associated with iPS. These include instabilities arising from the induced immature state and an increased risk of cancer if any pluripotent cells remain in the body.

The concept behind Mogrify actually predates, by nearly 19 years, Yamanakas discovery, which fast won him the 2012 Nobel Prize in Medicine. A 2017 Nature study on transdifferentiation, as the process is called, of fibroblasts into cardiac tissue traced the idea to a 1987 findingthat a master gene regulator could convert mice fibroblasts into skeletal muscle.

The problem though, according to Mogrify, is that most current efforts rely on an exhausting guess-and-check process. With hundreds of cell types and an even greater number of transcription factors the program that recodes the cell finding the right factor for the right cell can be like a custodian with a jangling, unmarked key ring trying to get into a building with thousands of locks.

Mogrifys key tech is a computer model they say can predict the right combination. The scientists behind the platform published a 2016 study in Nature applying the model to 173 human cell types and 134 tissues.

Before Mogrify, Disley led the Cambridge-based gene-editing company Horizon Discovery.

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Cell therapy startup raises $16 million to fund its quest for the Holy Grail in regenerative medicine - Endpoints News