Unravel Initiates RVL-001 Clinical Program in Colombia for Rett Syndrome With Clinical Trial Tool Translation

2024-02-13

临床研究

IRB Approval Received to Develop Validated Translations of Rett Syndrome Behavioral Questionnaire into Spanish, A Key Step for Clinical Trial Initiation

BOSTON, Feb. 13, 2024 /PRNewswire/ -- Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company to advance drugs for complex diseases, today announced the initiation of a clinical program in Colombia, starting with the translation of the widely-used Rett Syndrome Behavioral Questionnaire (RSBQ) into Spanish under an approved human subject research protocol. Participants are actively being recruited at https://www.unravel.bio/rsbq-translation-study.

Rett syndrome is a rare neurogenetic disorder starting in early childhood and leading to debilitating cognitive, motor and autonomic disability. With a global incidence of one in 10,000 female births, Rett Syndrome represents an orphan disease with widespread impact and only one FDA-approved treatment. Unravel's proprietary drug discovery platform has identified RVL001 as a promising therapeutic for Rett Syndrome, as well as RVL002, a novel small molecule therapeutic currently being developed. RVL001 will be tested in Colombia as a key path to effective treatment for Rett syndrome patients.

"The translation of the RSBQ represents a major expansion of Rett syndrome research and a key tool for clinical assessment to the more than 500 million Spanish speakers around the world," said Jeffrey Neul, MD, PhD, study co-Investigator and Director of the Vanderbilt Kennedy Center. "I look forward to working with Unravel to publish this critical resource and help facilitate their initiation of clinical research in Latin America."

"We are thrilled to collaborate with Unravel on this exciting first step toward initiating clinical trials in Colombia," expressed María Carrasquilla, Rett patient caregiver and Somos Rett Colombia member. "Engaging Rett families in the translation of the RSBQ enables them to participate in the establishment of therapeutic programs across Latin America."

About Unravel Biosciences

Unravel Biosciences is the first rapid prototyping therapeutics company, integrating AI systems biology computation with rapid in vivo screening and clinical validation of discovered targets with unprecedented efficiency. Unravel leverages its proprietary BioNAV™ platform combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases. Unravel's platform has led to four clinical trials starting in 2024. Unravel's platform developed RVL002, a first-in-class new small molecule targeting mitochondrial metabolism, and RVL027, a molecule targeting a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform access to foundation and biotech partners to accelerate and clinically derisk therapeutics. www.unravel.bio and www.rareshift.org.

SOURCE Unravel Biosciences, Inc.

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