Category Archives: Genetic Medicine

Partners to Provide Unique Expertise and Resources into Immune System Exposure, Patient Segmentation, and Target Identification

WALTHAM, Mass., March 31, 2022 /PRNewswire/ -- ImmuneID, a biopharmaceutical company that leverages existing antibody responses to rapidly unveil the complexities of the immune system and reveal pathways leading to precise, transformative therapies, today announced strategic partnerships with two world-class healthcare research organizations. These partnerships will significantly advance ImmuneID's work to comprehensively profile immune system responses, unveil new targets for drug development, identify clinical biomarkers and disease sub-populations, and determine the root cause of disease.

Both partnerships will leverage ImmuneID's aiSPIRE platform and demonstrate its ability to uncover highly specific patient and population insights and accurately segment patient subgroups by screening vast quantities of peptide-antibody interactions, while concurrently applying artificial intelligence (AI) to reveal immune system exposures and responses. These novel insights will provide ImmuneID with the foundation for target identification and precision therapiesthat address the most challenging conditions in autoimmunity and other serious diseases.

Icahn School of Medicine at Mount Sinai Collaboration

ImmuneID will collaborate with Judy Cho, MD, Dean of Translational Genetics and Director of The Charles Bronfman Institute for Personalized Medicine at the Icahn School of Medicine at Mount Sinai in New York, NY, to conduct an expansive study comparing antibody reactivity profiles across a large patient population. This study aims to generate a global profile of antibody binding specificities in patients with various autoimmune conditions including Sjogren's syndrome, systemic lupus erythematosus (SLE), lupus nephritis (LN) and scleroderma. As part of this partnership, ImmuneID will have access to Mount Sinai's BioMeBiobank,one of the largest collections of de-identified patient samples in the United States that connects a broad range of clinical, demographic, ancestral and environmental information, as well as electronic medical records, along with genetic information. This access will allow ImmuneID to conduct research and generate critical data used to analyze and sequence genetic material and study comparative long-term health outcomes.

University Hospital Erlangen Partnership

Through its partnership with University Hospital Erlangen, in Erlangen, Germany, ImmuneID will collaborate with Professor Jrg Distler, Research group leader at the Department of Internal Medicine Rheumatology and Immunology, on a comprehensive study of antibody reactive profiles present in patients with various autoimmune conditions. Dr. Distler and other leading experts of the department will work with ImmuneID to analyze the patient data to identify precise patient subgroups, potential diagnostic biomarkers, and therapeutic targets.

"We are honored to partner with Mount Sinai and University Hospital Erlangen, both prominent voices in the field of immune system research," said James S. Scibetta, Chief Executive Officer of ImmuneID. "These exciting partnerships represent an important step forward for ImmuneID to harness the power of our aiSPIRE platform to unveil immune reactivities that will enable us to clearly identify and discover disease drivers for disorders that have very limited and ineffective treatment options."

"Patient samples contain vital information to understand immune-driven diseases. We apply our aiSPIRE platform to uncover the information contained in the samples, generate comprehensive maps of the immune responses and, in so doing, deconvolute the complexity of the immune responses," said Annalisa D'Andrea, PhD, President and Chief Scientific Officer of ImmuneID. "These partnerships are fundamental to growing our large, proprietary database."

About ImmuneID

ImmuneID is a biopharmaceutical company dedicated to rapidly illuminating and untangling the complexities of the immune system, revealing the pathways leading to precise, transformative therapiesfor the most challenging conditions in autoimmunity and other serious diseases. ImmuneID was founded in 2020 by Longwood Fund and is headquartered in Waltham, Massachusetts. To learn more, please visit http://www.immuneidrx.com or follow us on LinkedIn.

About ImmuneID's aiSPIRE Platform

ImmuneID'saiSPIREplatform screens billions of antibody-target interactions while concurrently applying AI to comprehensively reveal the history of immune responses in each patient. ImmuneID combines this proprietary data with deep patient-specific clinical findings and private and public 'omics data to develop a better understanding of the biology of a disease, identify disease-driving pathways and meaningfully segment patient populations to discover novel clinical targets and predict personalized routes of disease progression.

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ImmuneID Announces Key Partnerships with Icahn School of Medicine at Mount Sinai and University Hospital Erlangen to Advance Novel Immune System...

A newly announced expansion allows for more eligible individuals to participate inthe community health research programIn Our DNA SC. Its offered by the Medical University of South Carolina, in collaboration with Helix, a leading population genomics company. The program provides genetic results for three health conditions:

Genomicsis the study of all of your genes, including how they interact with each other and your environment. Organizers want to make sure the people who sign up for testing through In Our DNA SC reflect the states population, which is more than 25% African American.

Marvella Ford, Ph.D., a professor in the College of Medicine and associate director of population sciences and cancer disparities at MUSC Hollings Cancer Center, called the announcement tremendous.

We have an opportunity now to provide access to genetic testing through In Our DNA SC, to everyone here in the state of South Carolina. We want to make sure that this opportunity is seen and embraced by racially, ethnically and socioeconomically diverse communities. We don't want anyone to be left out of this wonderful opportunity.

Ford said Hollings is already trying to reach more people through itsmobile health unit, which travels to rural areas to give mammograms and Pap smears, and its newcommunity health van, which offers vaccines to prevent HPV-related cancers.

In Our DNA SC is an important addition to those efforts. The program, introduced last September, was initially available as a pilot to existing patients in select locations, resulting in an enrollment of 1,800 people. As of last week, it is now open to enroll 100,000 people.

The testing involves saliva samples. Theres no charge to participants, and individuals and their doctor will receive a copy of the results. So will researchers trying to answer questions about how DNA affects health, but they wont know the results belong to a specific participant to protect privacy.

MUSC President David Cole, M.D., a cancer surgeon who spoke at the news conference announcing In Our DNA SCs expansion, has seen how empowering genetic information can be for patients. Embracing precision or personalized medicine is a necessary paradigm shift. It's going to be required to help define new pathways in terms of prevention and care delivery.

MUSC Health CEO Patrick Cawley, M.D., agreed. I'm excited about this program that will advance the science of medicine and present new options for patients in South Carolina - options like proactive preventive care plans that a patient can monitor for early symptoms and maybe even intervene early, he said.

It offers options like determining ahead of time medications that a patient may be genetically sensitive to. And that allows us to modify the dose for that patient, or maybe even avoid the medication entirely. It can also help solve diagnostic dilemmas. There's nothing more frustrating to a patient or a family than not knowing what is happening.

James Lu, M.D., Ph.D., CEO and co-founder of Helix, said MUSC is in good company. We're very focused on enabling large-scale genomics programs with leading health systems across the country who are dedicated to making genomics part of standard of care so that all patients across their health system can access the very best in precision medicine and make sure that all patients can have equal access.

Other institutions working with Helix include the Mayo Clinic, HealthPartners in Minnesota and Renown Health in Nevada. These are very much likeminded institutions that are thinking about genomics as part of standard of care. And MUSC is obviously a leader in bringing that to all patients, Lu said.

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Community health research program offering insight about genetic health risk expands across state - Medical University of South Carolina

Washington, DC, March 31, 2022 (GLOBE NEWSWIRE) -- The Alliance for Regenerative Medicine, the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies, today released its 2021 Annual Report, showcasing a year of significant advances and forecasting continued growth ahead.

The 2021 Report presents important milestones for the sector over the past year, including the approval of six new gene, cell, and tissue-based therapies, record sector investment, and promising early-stage clinical readouts. Highlights include the release of new clinical data supporting the use of gene editing directly in the body, powerful early evidence that cell and gene therapies can treat complex diseases with multiple genetic causes, and results that suggest these therapies may be able to reverse biological damage that has already occurred.

The exciting advancements of 2021 demonstrate that regenerative medicine is here and its not slowing down, said Janet Lambert, CEO of ARM. Regulatory decisions on 10 new therapies are expected this year, five of which are gene therapies for rare diseases making 2022 perhaps the most impactful year ever for gene therapy. More and more patients stand to benefit from the promise of these transformative treatments. But there is significant work ahead of us to ensure that health care systems are ready.

There are 2,406 ongoing trials globally, including 222 in Phase 3. Of these, 1,171 are industry-sponsored trials and 1,235 are trials sponsored by academic and government institutions.

Cancer is the sectors biggest target

The 2021 Annual Report shows that cancer, in both rare and prevalent forms, remains the largest target for regenerative medicine, representing 52% of trials globally. Cell-based immuno-oncology (cell-based IO) trials, a category that includes CAR-T therapies, make up the largest proportion of industry trials (41%), surpassing the number of cell therapy trials for the first time. The number of cell-based IO trials is likely to continue to grow, as more than half (57%) of phase 1 industry trials utilize this approach, compared to only about a quarter (22%) of phase 3 trials.

Solid tumors now make up nearly half (45%) of the cell-based IO therapies in the pipeline, with gastrointestinal cancer as the most common target, representing 23% of solid tumor trials.

Diseases of the Central Nervous System follow cancer as the second largest target (6%) for regenerative medicines globally. The most targeted diseases in this category are Parkinsons (21 ongoing trials), ALS (15), MS (12) and Alzheimers (10).

The pipeline is advancing for both prevalent and rare diseases

Today, nearly 60% of global regenerative medicine trials target prevalent conditions.

And the nature of prevalent diseases being targeted is evolving. Musculoskeletal disorders such as bone fractures, osteoarthritis, and sports injuries make up 23% of phase 3 trials targeting a prevalent disease, but only 7% of phase 1 trials. Other prevalent disease areas targeted include diabetes and related complications (54 ongoing trials) and central nervous system diseases such as Parkinsons (21). With phase 3 programs in indications including critical limb ischemia and congestive heart failure, the first approval of a gene therapy for a prevalent disease could be just a few years out.

Rare diseases make up 41% of the pipeline globally. Sickle cell disease is the top rare disease target with 39 ongoing trials. Other top indications for rare disease trials include Hemophilia (28), Retinitis Pigmentosa (26), ALS and Thalassemia (15 each). ALS is the only complex, polygenic disease in the top five.

Gene editing continues its rise

Gene editing continues to be an exciting frontier for the sector, with 41 gene editing trials ongoing globally, 14 of which are in phase 1 and 27 of which are in phase 2. 80% of these trials use CRISPR gene-editing technology, which first entered the clinic in 2019. More than half (23) of the 41 trials target cancer; 20 are hematological malignancies, and 3 are solid tumor. The first CRISPR therapy (and first gene editing therapy overall) could be approved as early as 2023, for sickle-cell disease.

Making regenerative medicine accessible is key to serving patients

The 2019 FDA prediction that it would approve 10-20 cell and gene therapies per year by 2025 is within reach, said Lambert. With the promise of regenerative medicine rapidly becoming a reality for patients, its critical that we continue to work diligently to ensure that these therapies are accessible to as many patients as possible.

Healthcare systems are accustomed to paying for conventional chronic care that treats symptoms and leads to the accrual of costs over long periods of time. Cell and gene therapies address the root causes of disease in a single dose, or a limited number of treatments, resulting in front-loaded costs that create accessibility barriers. Paving the way for innovative payment models that recognize the unique value of these therapies is a priority for the regenerative medicine sector, and ARM will continue to advocate for removing barriers to such arrangements.

About Alliance for Regenerative Medicine (ARM)

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 12-year history, ARM has become the voice of the sector, representing the interests of 425+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

Stephen Majors Alliance for Regenerative Medicine smajors@alliancerm.org

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Cell and Gene Therapies Poised to Disrupt Health Care Status Quo With Wave of New Treatments - The Bakersfield Californian

image:The ARDD Meeting 2022 will be hosted on August 29 - September 2, 2022 view more

Credit: Insilico Medicine Hong Kong Limited

March 31, 2022 -- Joris Deelen, Ph.D., will present the latest research on the topic Identification and functional characterisation of genetic variants linked to human longevity at the worlds largest annual Aging Research and Drug Discovery conference (9th ARDD). Dr. Joris Deelen is the Research Group Leader at the Max Planck Institute for Biology of Ageing.

"Previous research on the genetics of human longevity has shown that this trait is likely mainly determined by rare genetic variants. Therefore, the work in my group is currently focused on the functional characterisation of rare genetic variants identified in long-lived individuals. I recently obtained an ERC Starting Grant and a Longevity Impetus Grant to support this work." said Joris Deelen, Ph.D., Research Group Leader at the Max Planck Institute for Biology of Ageing.

Joris Deelen obtained his Ph.D. at the Leiden University Medical Center in the Netherlands in2014. In 2016, he joined the Max Planck Institute for Biology of Ageing as a postdoctoralresearcher where he was promoted to Research Group Leader in 2020. The work in his groupfocusses on the establishment of novel human aging studies in Cologne to determine theefficacy of previously identified biomarkers of aging in clinical studies. The main focus is onbiomarkers that have been identified in large-scale international collaborations of human studies using omics-based approaches, such as metabolomics.

The conference proceedings of the ARDD are commonly published in peer-reviewed journals with the talks openly available at http://www.agingpharma.org. Please review the conference proceedings for 2019, 2020 and 2021https://www.aging-us.com/article/203859/text .

Aging is emerging as a druggable condition with multiple pharmaceuticals able to alter the pace of aging in model organisms. The ARDD brings together all levels of the field to discuss the most pressing obstacles in our attempt to find efficacious interventions and molecules to target aging. The 2022 conference is the best yet with top level speakers from around the globe. Im extremely excited to be able to meet them in person at the University of Copenhagen in late summer. said Morten Scheibye-Knudsen, MD, Ph.D., University of Copenhagen.

Aging research is growing faster than ever on both academia and industry fronts. The ARDD meeting unites experts from different fields and backgrounds, sharing with us their latest groundbreaking research and developments. Our last ARDD meeting took place both offline and online, and it was a great success. I am particularly excited that being a part of the ARDD2022 meeting will provide an amazing opportunity for young scientists presenting their own work as well as meeting the experts in the field. said Daniela Bakula, Ph.D., University of Copenhagen.

Many credible biopharmaceutical companies are now prioritized aging research for early-stage discovery or therapeutic pipeline development. It is only logical to prioritize therapeutic targets that are important in both aging and age-associated diseases. The patient benefits either way. The best place to learn about these targets is ARDD, which we organize for nine years in a row. This conference is now the largest in the field and is not to be missed, said Alex Zhavoronkov, Ph.D., founder and CEO of Insilico Medicine and Deep Longevity.

Building on the success of the ARDD conferences, the organizers developed the Longevity Medicine course series with some of the courses offered free of charge at Longevity.Degree covered in the recent Lanced Healthy Longevity paper titled Longevity medicine: upskilling the physicians of tomorrow.

About Aging Research for Drug Discovery Conference

At ARDD, leaders in the aging, longevity, and drug discovery field will describe the latest progress in the molecular, cellular and organismal basis of aging and the search for interventions. Furthermore, the meeting will include opinion leaders in AI to discuss the latest advances of this technology in the biopharmaceutical sector and how this can be applied to interventions. Notably, this year we are expanding with a workshop specifically for physicians where the leading-edge knowledge of clinical interventions for healthy longevity will be described. ARRD intends to bridge clinical, academic and commercial research and foster collaborations that will result in practical solutions to one of humanity's most challenging problems: aging. Our quest? To extend the healthy lifespan of everyone on the planet.

About Scheibye-Knudsen Lab

In the Scheibye-Knudsen lab we use in silico, in vitro and in vivo models to understand the cellular and organismal consequences of DNA damage with the aim of developing interventions. We have discovered that DNA damage leads to changes in certain metabolites and that replenishment of these molecules may alter the rate of aging in model organisms. These findings suggest that normal aging and age-associated diseases may be malleable to similar interventions. The hope is to develop interventions that will allow everyone to live healthier, happier and more productive lives.

About Deep Longevity

Deep Longevity has been acquired by Edurance RP (SEHK:0575.HK), a publicly-traded company. Deep Longevity is developing explainable artificial intelligence systems to track the rate of aging at the molecular, cellular, tissue, organ, system, physiological, and psychological levels. It is also developing systems for the emerging field of longevity medicine enabling physicians to make better decisions on the interventions that may slow down, or reverse the aging processes. Deep Longevity developed Longevity as a Service (LaaS) solution to integrate multiple deep biomarkers of aging dubbed "deep aging clocks" to provide a universal multifactorial measure of human biological age. Originally incubated by Insilico Medicine, Deep Longevity started its independent journey in 2020 after securing a round of funding from the most credible venture capitalists specializing in biotechnology, longevity, and artificial intelligence. ETP Ventures, Human Longevity and Performance Impact Venture Fund, BOLD Capital Partners, Longevity Vision Fund, LongeVC, co-founder of Oculus, Michael Antonov, and other expert AI and biotechnology investors supported the company. Deep Longevity established a research partnership with one of the most prominent longevity organizations, Human Longevity, Inc. to provide a range of aging clocks to the network of advanced physicians and researchers. https://longevity.ai/

About Endurance RP (SEHK:0575.HK)

Endurance RP is a diversified investment group based in Hong Kong currently holding various corporate and strategic investments focusing on the healthcare, wellness and life sciences sectors. The Group has a strong track record of investments and has returned approximately US$298 million to shareholders in the 21 years of financial reporting since its initial public offering. https://www.endurancerp.com

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Joris Deelen to present at the 9th Aging Research & Drug Discovery Meeting 2022 - EurekAlert

In a paper published on March 11, 2022, in Sciences Advances, researchers in the Department of Chemistry and the Department of Physics & Astronomy at the University of California, Irvine revealed new details about a key enzyme that makes DNA sequencing possible. The finding is a leap forward into the era of personalized medicine when doctors will be able to design treatments based on the genomes of individual patients.

Enzymes make life possible by catalyzing chemical transformations that otherwise would just take too long for an organism, said Greg Weiss, UCI professor of chemistry and a co-corresponding author of the new study. One of the transformations were really interested in is essential for all life on the planet its the process by which DNA is copied and repaired.

This image shows the Taq enzyme in its open configuration waiting for a new chemical base to arrive so it can try to fit it to a DNA chain. Credit: Max Strul / UCI and Lorena Beese laboratory / Duke University

The molecule the UCI-led team studied is an enzyme called Taq, a name derived from the microorganism it was first discovered in, Thermos aquaticus. The molecule the UCI-led team studied is an enzyme called Taq, a name derived from the microorganism it was first discovered in, Thermos aquaticus. Taq replicates DNA. Polymerase chain reaction, the technique with thousands of uses from forensics to PCR tests to detect COVID-19, takes advantage of Taq.

The UCI-led team found that Taq, as it helps make new copies of DNA, behaves completely unlike what scientists previously thought. Instead of behaving like a well-oiled, efficient machine continuously churning out DNA copies, the enzyme, Weiss explained, acts like an indiscriminate shopper who cruises the aisles of a store, throwing everything they see into the shopping cart.

Instead of carefully selecting each piece to add to the DNA chain, the enzyme grabs dozens of misfits for each piece added successfully, said Weiss. Like a shopper checking items off a shopping list, the enzyme tests each part against the DNA sequence its trying to replicate.

Its well-known that Taq rejects any wrong items that land into its proverbial shopping cart that rejection is the key, after all, to successfully duplicating a DNA sequence. Whats surprising in the new work is just how frequently Taq rejects correct bases. Its the equivalent of a shopper grabbing half a dozen identical cans of tomatoes, putting them in the cart, and testing all of them when only one can is needed.

The take-home message: Taq is much, much less efficient at doing its job than it could be.

The find is a leap toward revolutionizing medical care, explained Philip Collins, a professor in the UCI Department of Physics & Astronomy whos a co-corresponding author of the new research. Thats because if scientists understand how Taq functions, then they can better understand just how accurate a persons sequenced genome truly is.

Every single person has a slightly different genome, said Collins, with different mutations in different places. Some of those are responsible for diseases, and others are responsible for absolutely nothing. To really get at whether these differences are important or healthcare for properly prescribing medicines you need to know the differences accurately.

Scientists dont know how these enzymes achieve their accuracy, said Collins, whose lab created the nano-scale devices for studying Taqs behavior. How do you guarantee to a patient that youve accurately sequenced their DNA when its different from the accepted human genome? Does the patient really have a rare mutation, asks Collins, or did the enzyme simply make a mistake?

This work could be used to develop improved versions of Taq that waste less time while making copies of DNA, Weiss said.

The impacts of the work dont stop at medicine; every scientific field that relies on accurate DNA sequencing stands to benefit from a better understanding of how Taq works. In interpreting evolutionary histories using ancient DNA, for example, scientists rely on assumptions about how DNA changes over time, and those assumptions rely on accurate genetic sequencing.

Weve entered the century of genomic data, said Collins. At the beginning of the century we unraveled the human genome for the very first time, and were starting to understand organisms and species and human history with this newfound information from genomics, but that genomic information is only useful if its accurate.

Reference: Single-molecule Taq DNA polymerase dynamics by Mackenzie W. Turvey, Kristin N. Gabriel, Wonbae Lee, Jeffrey J. Taulbee, Joshua K. Kim, Silu Chen, Calvin J. Lau, Rebecca E. Kattan, Jenifer T. Pham, Sudipta Majumdar, Davil Garcia, Gregory A. Weiss and Philip G. Collins, 11 March 2022, Science Advances.DOI: 10.1126/sciadv.abl3522

Co-authors on this study include Mackenzie Turvey, Ph.D., a former UCI graduate student in physics & astronomy, and Kristin Gabriel, Ph.D., a former UCI graduate student in molecular biology & biochemistry. This research was funded by the National Human Genome Research Institute of the NIH.

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Leap Forward in Genetic Sequencing Will Lead to Improved Personalized Medicine and Understanding of Evolution - SciTechDaily

NEW YORK Earlier this month, the U.S. Patent and Trademark Office ruled that CRISPR patents key to developing human therapies belong to the Broad Institute of MIT and Harvard, ending the latest chapter in a bitter seven-year battle between the Broad and the home institutions of Jennifer Doudna and Emmanuelle Charpentier the two scientists who won the Nobel Prize for creating the revolutionary gene-editing technology.

But for all the acrimony exchanged and millions of dollars of legal fees spent by the academic institutions where CRISPR was first invented, the companies that are actually turning the technology into medicines are plowing through the fallout of the decision with little more than a collective shrug.

I think its really a topic for people interested in IP law, Lawrence Klein, the chief operating officer of CRISPR Therapeutics said Thursday during the 2022 STAT Breakthrough Science Summit in New York City.

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Klein said the ruling isnt changing the companys focus or its commercialization strategy. At the end of the day, there might be a modest economic impact one way or another, but when you zoom out theres so much exciting science and innovation going on thats going to improve peoples lives, he said. Its just not something to focus on, in my opinion.

Laura Sepp-Lorenzino, executive vice president and chief scientific officer at Intellia Therapeutics, was quick to concur. Fully agree, she said.

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To develop their pipelines of gene editing treatments, Intellia and CRISPR Therapeutics both licensed patents from the University of California. CRISPR Therapeutics has had early clinical success treating inherited blood conditions, and Intellia recently announced that a one-time treatment of its therapy for a genetic nerve disorder lowered levels of the disease-causing protein for up to nine months the first biotech company to report success with a so-called in vivoCRISPR-based medicine.

Editas Medicine, which for now holds a competitive intellectual-property advantage with its exclusive license on applications of Broad scientist Feng Zhangs patents for treating human disease, has fallen behind its rivals in the push to market. After a series of setbacks, a clinical trial of its treatment for genetic blindness finally began last year, but so far has shown mixed results.

As things stand now, Editas could, in theory, seek to block competitors from selling CRISPR-based medicines, if and when they receive Food and Drug Administration approval to do so. Such a scenario is rare, but it has happened before. In 2014, Amgen sued its partners Sanofi and Regeneron over alleged patent infringement on its PCSK9 inhibitor, Repatha, successfully scuttling the drugs launch in the U.S. market. But far more routine is for companies to sort out licensing deals that benefit them both.

On Thursday, Editas leadership seemed to indicate, more strongly than it has in previous public statements, that the latter is the likely way this will play out for CRISPR.

It is never going to be our intention to prevent any of the other gene editing companies from bringing a therapy to the market, said Mark Shearman, Editas executive vice president and chief scientific officer. Im sure at some point in the future the legal teams across the organizations will figure this out.

Still, much remains up in the air, even as the intellectual property around CRISPR is growing increasingly complex. Patent offices in other countries have reached different decisions about who invented what. And additional parties have entered the fray ToolGen in South Korea and Sigma-Aldrich, owned by Merck KGaA in Germany, are challenging the ownership of some of the early, foundational CRISPR patents, legal disputes that could take years to play out while further embroiling both the Broad and the University of California, and the companies that licensed their gene-editing technology.

Decisions down the road could shift the landscape yet again. And meanwhile, no one wants to slow down the push toward developing what could be one-time cures for long-neglected diseases.

I think well be talking around the table, Sepp-Lorenzino said, addressing Shearman to a ripple of timid laughter from the audience. Because you need some of our patents, and who knows, maybe we need one of yours. Or maybe not, so well see.

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In the wake of big patent decision, it's business as usual for CRISPR therapy developers - STAT