RVL-027

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

New study will use Unravel's AI platform to stratify patients into subgroups based on their likelihood to respond to prioritized drugs identified in earlier phase of research. BOSTON, Aug. 28, 2024 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company established to advance drugs for complex diseases, and the CSNK2A1 Foundation ("Foundation") today announced that they are expanding their drug discovery collaboration to now stratify Okur-Chung Neurodevelopmental Syndrome (OCNDS) patients into subgroups based on their predicted response to selected drugs. Through a previously announced collaboration, Unravel's proprietary BioNAV™ drug discovery platform used RNA data from patient-derived cells and mouse models to identify in silico drug hits. The Unravel study revealed that patient response to predicted drugs may vary significantly based on the location of their specific CSNK2A1 gene mutation causing OCNDS. The Foundation independently confirmed several in silico hits in a Drosophila fruit fly animal model of OCNDS that have the potential for clinical benefit through a screen with Dr. Clement Chow at the University of Utah. As part of its rareSHIFT™ discovery services, Unravel will utilize its proprietary nasal swab collection methodology in the new project to collect RNA transcriptomes from OCNDS patients and matched healthy family members. The data will be processed using BioNAV™ to identify drug candidates most likely to work for patients with specific genetic mutations. This project focuses on the most prevalent OCNDS-causing mutation, K198R, located in the activation segment of CSNK2A1. Currently the Foundation has over 70 individuals registered with this specific variant. "Given that even monogenetic disorders have substantial variability in how they clinically present and respond to drugs, having a data-driven preview of drug response in real patients is essential to clinically derisk preclinical programs like this," said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "By matching each patient to a drug candidate, we will increase the probability of success in clinical trials and reduce the time and cost of developing meaningful therapies for OCNDS patients." "This innovative approach allows us to move beyond a one-size-fits-all model, offering the possibility of more personalized and effective therapies for our community," explained Gabrielle Rushing, Ph.D., CSNK2A1 Foundation Science Program Director. "The insights gained from stratifying patients by their specific genetic mutations will be invaluable in our ongoing mission to accelerate the development of meaningful treatments for OCNDS." First described in 2016, OCNDS is an ultra-rare genetic syndrome caused by a mutation on chromosome 20 in the CSNK2A1 gene. OCNDS affects every aspect of life, with symptoms ranging from mild to severe and can include the inability to speak or delayed speech, global developmental delay, epilepsy, autism spectrum disorder traits, behavioral challenges, hypotonia, feeding difficulty, and severe sleep disruption. To date, there is no treatment for OCNDS. While the overall prevalence of OCNDS is not fully known, over 250 individuals worldwide are currently registered with the Foundation. 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 CSNK2A1 Foundation CSNK2A1 Foundation is focused on finding a cure for Okur-Chung Neurodevelopmental Syndrome and ensuring affected individuals have the opportunities and supports necessary for happy and full lives. CSNK2A1 Foundation is a 501(c)(3) non-profit organization that is operated and funded by a committed team of volunteers, advocates, and researchers. To date, the foundation has registered over 250 individuals with OCNDS in its patient registry worldwide. SOURCE Unravel Biosciences, Inc.