Unravel Biosciences Announces Approval by Colombian Health Regulatory Authority to Initiate RVL-001 Clinical Studies for Rett Syndrome and Pitt Hopkins Syndrome

Audio By Carbonatix

BOSTON--(BUSINESS WIRE)--Dec 2, 2025--

Unravel Biosciences, Inc., (“Unravel”), a clinical-stage therapeutics company established to advance drugs for complex diseases through its Predictable Medicine™ platform, today announced the approval to initiate Unravel’s RVL-001 clinical trials for Rett syndrome (“RTT”) and Pitt Hopkins syndrome (“PTHS”) in Colombia by the Colombian Health Regulatory Agency (“INVIMA”).

Unravel's proprietary data and BioNAV™ discovery engine established dynamic transcriptome network profiles, called “Living Molecular Twins,” to identify RVL-001 as a potentially promising therapeutic drug for RTT and PTHS. RVL-001 targets a novel therapeutic mechanism, which will be clinically evaluated for the first time in these studies. In addition to Unravel’s RVL-001 program for RTT and PTHS, the company has also initiated development of RVL-002, a first-in-class novel molecule for RTT syndrome.

INVIMA approval allows both programs to commence recruiting immediately, with first patient dosing anticipated in January 2026 at the Universidad de Antioquia’s Center for Technological Development (“PECET”), a clinical study center of excellence and designated INVIMA clinical trial site in Medellin, Colombia. Both RVL-001 proof of concept studies are placebo-controlled, “n-of-1” trials with a target enrollment of 15 RTT patients and 5 PTHS patients. More information can be found at https://clinicaltrials.gov/study/NCT07150013 and https://clinicaltrials.gov/study/NCT07150026

"We are thrilled to commence both clinical trials as a critical milestone in our patient-driven, platform-developed therapeutics programs,” said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "Today’s announcement is a validation of our efficient approach towards rapid clinical testing of promising therapeutics for rare and complex disorders, conditions that afflict nearly 10% of the world’s population."

RTT and PTHS are both rare neurogenetic disorders starting in early childhood that lead to debilitating cognitive, motor and autonomic disability. Despite one approved treatment for RTT, there remains a significant unmet need for novel treatments having meaningful efficacy and acceptable safety and tolerability. There are no known treatments for PTHS.

About Unravel Biosciences

Unravel Biosciences is a clinical-stage therapeutics company that integrates AI systems biology computation with rapid clinical validation of discovered targets, leading to four clinical trials starting in 2026. Unravel leverages its proprietary BioNAV™ platform and primary transcriptomics data to create Living Molecular Twins of real patients that can be used to predict therapeutic response. This enables target and drug discovery, preclinical screening, patient stratification, and clinical validation to find treatments for complex diseases using a systematic, data-driven approach called Predictable Medicine™. Unravel's platform discovered RVL002, a first-in-class small molecule that targets mitochondrial metabolism and has multiple clinical applications, including neurodegenerative and metabolic disorders, and identified RVL069, a molecule that targets a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform and proprietary datamine access to foundation and biotech partners to accelerate and clinically derisk therapeutics. www.unravel.bio and www.rareshift.org

View source version on businesswire.com:https://www.businesswire.com/news/home/20251202077465/en/

CONTACT: Unravel Biosciences, Inc.

[email protected]

+1 510-984-2317

KEYWORD: MASSACHUSETTS LATIN AMERICA NORTH AMERICA UNITED STATES SOUTH AMERICA COLOMBIA

INDUSTRY KEYWORD: TECHNOLOGY RESEARCH CLINICAL TRIALS SOFTWARE BIOTECHNOLOGY HEALTH PHARMACEUTICAL SCIENCE ARTIFICIAL INTELLIGENCE

SOURCE: Unravel Biosciences, Inc.

Copyright Business Wire 2025.

PUB: 12/02/2025 08:30 AM/DISC: 12/02/2025 08:30 AM

http://www.businesswire.com/news/home/20251202077465/en