Unravel Biosciences and PECET (the clinical research unit of the University of Antioquia) have finalized a clinical trial agreement to evaluate RVL-001 as a potential treatment for Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS). PECET, certified in Good Clinical Practices, will serve as the designated study site in Medellin, Colombia.
Clinical Trial Details
The collaboration aims to accelerate the development of therapeutics for these rare neurogenetic disorders. Unravel's proprietary BioNAV™ drug discovery platform identified RVL-001 as a promising therapeutic candidate. The trials are set to begin in early 2025, with study startup activities already underway, including clinical trial material manufacturing and patient recruitment.
Addressing Unmet Needs in Rare Diseases
RTT and PTHS are rare neurogenetic disorders that manifest in early childhood, leading to significant cognitive, motor, and autonomic disabilities. While there is one approved treatment for RTT, substantial unmet needs remain for therapies with improved efficacy and safety profiles. Currently, there are no approved treatments for PTHS.
Richard Novak, Ph.D., Co-Founder and CEO of Unravel, stated, "By conducting multiple clinical trials in parallel at a single clinical trial site enables faster and less costly drug development to support more patients with unmet needs."
RVL-001 and RVL-002
In addition to RVL-001, Unravel Biosciences has initiated development work on RVL-002, a first-in-class novel molecule for Rett syndrome, further expanding their pipeline of potential treatments for these challenging conditions.
Iván Darío Vélez, Professor Emeritus of the University of Antioquia and founder of PECET, commented, "PECET has extensive expertise conducting clinical studies with international biopharma sponsors including US to support worldwide registration and approval and we look forward to supporting Unravel in their development efforts".
