Unravel Biosciences: AI-Driven Drug Repurposing Trial

RVL-001 Advances Toward Clinical Trials

Unravel Biosciences, an AI-enabled therapeutics company, is preparing to initiate a proof-of-concept clinical trial for RVL-001, a potential treatment for Pitt Hopkins Syndrome (PTHS), in Colombia. RVL-001 is a repurposed formulation of vorinostat, a histone deacetylase inhibitor previously identified by Drs. Andrew Kennedy and Dr. David Sweatt, PHRF-funded researchers. Unravel’s proprietary BioNAV™ platform identified RVL-001 as a promising candidate for PTHS as well as Rett Syndrome.

Histone deacetylases are a type of enzyme that helps to control how tightly DNA is wrapped around histones. By blocking these enzymes, histone deacetylase inhibitors can act to modulate the activity of genes within cells.

The trial is expected to enroll 6 people with Pitt Hopkins syndrome and will test the experimental therapy against a placebo and will be conducted in collaboration with PECET, the clinical research unit at the University of Antioquia in Medellín, Colombia. PECET is certified in Good Clinical Practices by Colombia’s health regulatory agency, INVIMA. The study has been submitted under INVIMA’s pilot program, which allows for priority review and fast-track approval for orphan diseases and conditions with high unmet medical needs.

This initiative builds upon Unravel’s prior efforts in Colombia, where infrastructure was established for a Rett Syndrome trial. The Pitt Hopkins Research Foundation is funding the PTHS trial and patient enrollment for the RVL-001 trial is anticipated to begin by August 2025. This collaborative effort exemplifies the potential of repurposing existing drugs to address rare diseases.