The Jackson Laboratory

The collaboration will allow for a more rapid and efficient approach to the identification and pre-clinical evaluation of potential drug candidates across multiple diseases to advance into clinical testing. BOSTON and BAR HARBOR, Maine, Sept. 17, 2024 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company spun out of Harvard University to advance drugs for complex diseases and The Jackson Laboratory (JAX), a pioneer in the discovery of precise genomic solutions for disease, today announced the initiation of a collaboration to expedite and efficiently identify promising drug candidates for clinical development. Unravel will utilize their proprietary BioNAV™ drug discovery platform and JAX's extensive expertise in pre-clinical disease modeling to identify potential drugs and novel drug targets across multiple diseases. The collaboration will prioritize already approved drugs that have the potential to be repurposed for other diseases, facilitating rapid clinical translation and near-term beneficial impact to patients. "We are thrilled to collaborate with JAX on this key multi-program effort to efficiently tackle multiple rare disorders," said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "By seamlessly iterating between preclinical models and advanced computational analyses of multimodal datasets, we are revolutionizing the time, cost, and risk of drug discovery to enable rapid clinical translation and derisking for patients in need." The first disease to be studied in the collaboration will be Alternating Hemiplegia of Childhood (AHC), a rare neurodevelopmental disorder characterized by repeated episodes of weakness or paralysis leading to hemiplegia or quadriplegia. AHC is estimated to occur in approximately 1 in 1,000,000 births and is caused by mutations in the ATP1A3 gene. Unravel will generate RNASeq data from JAX's ATP1A3 disease relevant mouse models and analyze the data using Unravel's BioNAV™ platform to generate in silico hits of promising therapeutic candidates. These drug candidates will then be evaluated in JAX's pre-clinical models for efficacy as well as to inform dose selection for clinical testing. "Our collaboration with Unravel Biosciences is deeply meaningful for the patients and foundations we serve," said Cat Lutz, Ph.D., MBA, vice president of the Rare Disease Translational Center at JAX. "Validating Unravel's leads in our patient-specific mouse models has the potential to provide immediate benefits to patients and pave the way for future targeted genetic therapies. We also believe linking our scientific resources creates more seamless navigation for patients and families." While it is expected that rare disease foundations will drive much of the initial demand for research under the collaboration, this innovative drug discovery work is also anticipated to generate interest by biotechnology and pharmaceutical companies to support their drug development programs, as well as for use by Unravel and JAX to accelerate development of their own proprietary assets. About Unravel Biosciences Unravel Biosciences is the first rapid prototyping therapeutics company, integrating AI systems biology computation with rapid in vivo screening and clinical validation of discovered targets with unprecedented efficiency. Unravel leverages its proprietary BioNAV™ platform combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases. Unravel's platform has led to four clinical trials starting in 2024. Unravel's platform developed RVL002, a first-in-class new small molecule targeting mitochondrial metabolism, and RVL027, a molecule targeting a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform access to foundation and biotech partners to accelerate and clinically derisk therapeutics. and About The Jackson Laboratory The Jackson Laboratory is an independent, nonprofit biomedical research institution with a National Cancer Institute-designated Cancer Center and more than 3,000 employees in locations across the United States, Japan and China. Its mission is to discover precise genomic solutions for disease and empower the global biomedical community in the shared quest to improve human health. For more information, please visit . SOURCE Unravel Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

This recognition by the Society is its highest honor in the academic fields of arts, social sciences, humanities and sciences. FARMINGTON, Conn., Sept. 11, 2024 /PRNewswire/ -- Charles Lee, PhD, DSc, FACMG, has been elected to the Royal Society of Canada (RSC) incoming class of 2024 Fellows, in recognition of his outstanding achievements in the field of human genetics. The RSC is the senior national body of distinguished Canadian scientists and scholars and the oldest scientific academy in continuous existence in Canada. At The Jackson Laboratory for Genomic Medicine, where he is Professor and the Robert Alvine Family Endowed Chair, Lee heads a lab that uses state-of-the-art technologies to study structural genomic variation and its contributions to human diseases and vertebrate genome evolution. This prestigious designation is the Society's highest honor, presented to scholars in the arts, social sciences, humanities and sciences for their invaluable contributions to their fields. This year's class of 104 new Fellows were elected by their peers for their exceptional contributions in these fields. Lee is one of 51 Fellows elected to the Academy of Sciences this year. "I am overjoyed to be chosen as a Royal Society of Canada Fellow," said Lee. "The scientific accomplishments we have been able to achieve in my lab were possible only because of the collaboration and research support from many institutions, including the University of Alberta and Natural Sciences and Engineering Research Council of Canada. I am inspired by this year's class of Fellows and honored to be counted among them." Lee is known for his discoveries in human genetics, including a landmark 2004 paper that detected widespread structural changes within the genome, which transformed the understanding of disease mechanisms and human evolution. Most recently, Lee's laboratory completed the very first complete genetic sequencing of human Y chromosomes. Lee has written more than 190 publications which have collectively been cited more than 100,000 times. Lee grew up in High Prairie, Alberta, Canada and subsequently studied at the University of Alberta. In 2013, he joined JAX from Harvard Medical School and Brigham and Women's Hospital. Throughout his career, he has received numerous accolades and awards for his research into the human genome, including the 2008 Ho-Am Prize in Medicine and was a Thomson Reuters Citation Laureate in 2012. He is an elected fellow of the American Association for the Advancement of Science (AAAS) and a fellow of the Korean Academy of Science and Technology. He previously served as the president of the Human Genome Organization (HUGO) from 2017-2023 and received an honorary doctorate of science from the University of Alberta, his alma mater, earlier this year. Founded in 1882, the Royal Society of Canada is an independent scientific academy. Its Fellows have included many of the world's most eminent scientists including Sir Sandford Fleming – the originator of the worldwide standard time zones – Joseph Burr Tyrrell – a Canadian geologist who discovered a new dinosaur species in the Alberta Badlands – and John Lighton Synge – who introduced a new geometric approach to the theory of relativity and helped establish the Fields Medal. The new Fellows will be inducted into the Society during a ceremony on November 8, 2024, in Vancouver, British Columbia. About The Jackson Laboratory The Jackson Laboratory is an independent, nonprofit biomedical research institution with a National Cancer Institute-designated Cancer Center and more than 3,000 employees in locations across the United States, Japan and China. Its mission is to discover precise genomic solutions for disease and to empower the global biomedical community in the shared quest to improve human health. For more information, please visit . About the Royal Society of Canada Founded in 1882, the Royal Society of Canada (RSC) comprises The Academy of Arts and Humanities, The Academy of Social Sciences, The Academy of Science, and The RSC College. The RSC recognizes excellence, advises the government and the larger society, and promotes a culture of knowledge and innovation in Canada and with other national academies around the world. Learn more at . SOURCE The Jackson Laboratory WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Our bone marrow the fatty, jelly-like substance inside our bones is an unseen powerhouse quietly producing 500 billion new blood cells every day. That process is driven by hematopoietic stem cells that generate all of the various types of blood cells in our bodies and regenerating themselves to keep the entire assembly line of blood production operating smoothly. Our bone marrow -- the fatty, jelly-like substance inside our bones -- is an unseen powerhouse quietly producing 500 billion new blood cells every day. That process is driven by hematopoietic stem cells that generate all of the various types of blood cells in our bodies and regenerating themselves to keep the entire assembly line of blood production operating smoothly. As with any complex system, hematopoietic stem cells lose functionality as they age -- and, in the process, contribute to the risk of serious diseases, including blood cancers. We know that the risk of developing aging-associated diseases is different among different individuals. Surprisingly, however, little is known about whether hematopoietic stem cells age differently between individuals. "If you take a room full of 50-year-olds, some will be completely gray-haired, others will be salt-and-pepper, and a few will not have any gray hairs at all," said Jennifer Trowbridge, Dattels Family Endowed Chair and professor at the Jackson Laboratory. "Logically, you'd expect to see the same kind of variation in the function of hematopoietic stem cells -- but until now, nobody has studied that directly." For good reason: Because these hematopoietic stem cells are so rare, researchers typically pool all of these stem cells together, studying them in aggregate. In a paper published in Blood, Trowbridge and colleagues studied hematopoietic stem cells at the single cell level in nine individual, genetically identical middle-aged mice -- offering the first close look at how subtle changes in the bone marrow microenvironment ages hematopoietic stem cells across individual mice. Trowbridge and team found that despite the mice being all the same age, the hematopoietic stem cells in the bone marrow of these individual mice aged differently. But that's not all. The team could predict the function of the hematopoietic stem cells based on the activity of two growth factors that are also present in humans. The two growth factors -- Kitl and Igf1 -- are produced by mesenchymal stromal cells (MSC) that surround the stem cells in the bone marrow microenvironment. By profiling the RNA transcriptome in these MSCs across individual mice, Trowbridge found that the decline of these growth factors correlated with age-associated molecular programs in hematopoietic stem cells. "The amount of the growth factors that are being produced directly correlates to the declining function of the stem cells -- and we found markedly more variation in hematopoietic stem cells than in other cells in the bone marrow," Trowbridge said. "This is really a snapshot of the aging process at work, at the cellular level." " In humans, who are genetically diverse and have varying lifestyles, variations in hematopoietic stem cell aging are likely to be even greater than in carefully controlled animal models, explained Trowbridge. While the current study didn't explore whether cellular aging of the stem cells directly triggers adverse health outcomes, it's likely that such variations play a role in a wide range of health outcomes for both mice and humans.