Unravel Biosciences and The Jackson Laboratory Collaborate to Speed Up Drug Discovery for High-Need Diseases

Unravel Biosciences, an AI-driven therapeutics firm spun out from Harvard University, has teamed up with The Jackson Laboratory (JAX), a leader in genomic solutions for diseases, to accelerate the discovery and pre-clinical evaluation of drug candidates for various diseases. This partnership aims to quickly identify and develop promising drugs for clinical use.

The collaboration will leverage Unravel's BioNAV™ drug discovery platform alongside JAX's extensive expertise in pre-clinical disease modeling. A key focus will be on repurposing already approved drugs for new disease indications, thereby speeding up the clinical translation process and providing quicker benefits to patients.

Richard Novak, Ph.D., Co-Founder and CEO of Unravel, emphasized the importance of this collaboration in addressing multiple rare disorders efficiently. He highlighted how the integration of preclinical models with advanced computational analyses allows for a significant reduction in the time, cost, and risk associated with drug discovery, ultimately enabling faster clinical applications.

The first target of this partnership will be Alternating Hemiplegia of Childhood (AHC), a rare neurodevelopmental disorder that causes recurrent episodes of weakness or paralysis. This condition, affecting about 1 in 1,000,000 births, is linked to mutations in the ATP1A3 gene. Unravel will produce RNASeq data from JAX's mouse models relevant to the ATP1A3 gene and use the BioNAV™ platform to identify potential therapeutic candidates in silico. These candidates will then undergo efficacy testing in JAX's pre-clinical models and will be used to determine appropriate doses for clinical trials.

Cat Lutz, Ph.D., MBA, vice president of the Rare Disease Translational Center at JAX, expressed enthusiasm about the collaboration's potential to benefit patients directly. By validating Unravel's leads in patient-specific mouse models, the partnership aims to lay the groundwork for future targeted genetic therapies. Lutz also mentioned that integrating scientific resources from both organizations would facilitate better navigation for patients and their families.

The primary interest in this collaboration is expected to come from rare disease foundations, but it is also anticipated to attract biotechnology and pharmaceutical companies. These companies may seek to support their drug development programs using the innovative work produced from this partnership. Additionally, Unravel and JAX aim to use the generated insights to advance their proprietary assets.

Unravel Biosciences combines AI systems biology computation with rapid in vivo screening and clinical validation. Their BioNAV™ platform is central to their operations, offering an integrated approach to target and drug discovery, preclinical screening, and patient stratification. This platform has already led to the initiation of four clinical trials in 2024. Two notable developments from Unravel include RVL002, a small molecule targeting mitochondrial metabolism, and RVL027, which targets a novel mechanism to treat dystonias. The company's rareSHIFT™ program provides access to their platform for foundation and biotech partners, helping to accelerate and reduce the risk of therapeutic development.

The Jackson Laboratory is a nonprofit biomedical research institution with an NCI-designated Cancer Center, employing over 3,000 people across the United States, Japan, and China. Their mission is to discover precise genomic solutions for diseases and support the global biomedical community in improving human health.