Unravel Partners with Vanderbilt for Rett Syndrome Study Including RVL-001

Unravel Biosciences, Inc., an AI-driven therapeutics firm, has announced a partnership with Vanderbilt University Medical Center (VUMC) to support a multisite clinical trial aimed at identifying treatment options for Rett Syndrome using repurposed FDA-approved drugs. The trial will include an oral liquid formulation of vorinostat, known as RVL-001, which was predicted by Unravel's BioNAV platform to be a potential treatment for the disease.

Funded by a $13 million Department of Defense grant, the study will compare three drugs—ketamine, vorinostat, and donepezil—against a placebo to evaluate their safety, tolerability, efficacy, and biomarker responses over a 16-week period. Participants will be randomly assigned to one of four treatment groups.

In this collaboration, Unravel will provide essential analytical and formulation support for vorinostat (RVL-001) and assist VUMC's clinical and regulatory teams. In return, Unravel will gain access to clinical data generated from the vorinostat trials, aiding its own development and commercialization efforts.

Richard Novak, Ph.D., Co-Founder and CEO of Unravel, expressed enthusiasm about the collaboration, stating, "Our participation in this landmark study led by Dr. Jeff Neul at VUMC validates the potential of our BioNAV platform technology." Unravel’s platform identified RVL-001 as a promising candidate for treating Rett Syndrome, a rare neurogenetic disorder affecting cognitive, motor, and autonomic functions that predominantly impacts one in every 10,000 female births. Despite its significant impact, there is currently only one FDA-approved treatment for the condition.

Jeffrey Neul, M.D., Ph.D., Director of the Vanderbilt University Kennedy Center and the study’s Principal Investigator, emphasized the importance of industry support in advancing the development and commercialization of new therapies for Rett Syndrome. He highlighted that such collaborations benefit both patients and the clinical community.

Unravel Biosciences is pioneering a new approach to therapeutics through its integration of AI systems biology with rapid in vivo screening and clinical validation. Their proprietary BioNAV platform facilitates target and drug discovery, preclinical screening, and patient stratification. This innovative platform has already led to the initiation of four clinical trials in 2024. Among the drugs developed by Unravel is RVL-002, a small molecule targeting mitochondrial metabolism, and RVL-027, designed to treat dystonias.

The company also runs the rareSHIFT™ program, which offers platform access to foundations and biotech partners, accelerating the development and reducing the clinical risk of new therapeutics. This program underscores Unravel's commitment to addressing complex diseases through advanced technology and collaborative research.

The clinical trial at VUMC, supported by Unravel, represents a significant step forward in the search for effective treatments for Rett Syndrome. By evaluating repurposed drugs like vorinostat, the study aims to provide new hope for patients suffering from this debilitating condition. The collaboration exemplifies the power of combining cutting-edge technology with clinical expertise to tackle challenging medical issues.