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 -- 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. www.unravel.bio and www.rareshift.org
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 www.jax.org.
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