Unravel Biosciences And COMBINED Brain Announce Large-Scale Drug Discovery Collaboration For Rare Neurological Diseases With High Unmet Need

The agreement represents one of the largest biopharma-patient community partnerships of its kind and will accelerate the identification of potential drug candidates across multiple ultra-rare diseases.

Boston, MA and Nashville TN /PRNewswire/ - Unravel Biosciences, Inc., ("Unravel"), an AI-enabled therapeutics company spun out of Harvard University to advance drugs for complex diseases and COMBINED Brain, a non-profit consortium devoted to speeding the path to clinical treatments for people with rare genetic neurological disorders by pooling efforts, studies, and data, today announced the initiation of a collaboration to identify promising drug candidates for clinical development across the more than 110 disorders represented by member patient advocacy groups in COMBINED Brain.

Unravel will utilize their proprietary BioNAV™ drug discovery platform and COMBINEDBrain's unique large-scale patient biosample repository and collection program to generate RNA datasets and identify potential drugs and novel drug targets across the entire spectrum of rare and ultra-rare patients. By tackling over 100 disorders in parallel, the collaboration will prioritize shared therapeutic mechanisms together with already approved drugs, facilitating rapid clinical translation and near-term beneficial impact to many patients.

"At COMBINED Brain, our priority is to get treatments to kids who need them as quickly as possible. For rare neurogenetic disorders, identification of promising new uses for approved drugs can be the fastest way to help, with several such examples of drugs making meaningful improvements for kids with severe epilepsy and developmental delays. This project is a way to identify drugs which we could pilot in clinical trials across multiple rare pediatric diseases at the same time" said Terry Jo Bichell, PhD, COMBINED Brain Chief Executive Officer.

"We are thrilled to collaborate with COMBINED Brain and their vast community of patients and their families," said Frederic Vigneault, PhD, Unravel Co-Founder and Chief Science Officer. "We share a sense of urgency and the belief that no one is too rare. We both recognize that by looking across patients and conditions, we can fast-track access to treatments."

The resulting datasets and drug candidates will accelerate the efforts of member patient advocacy groups to identify promising drugs, with the overall to identify shared therapeutic mechanisms across diseases that can be targeted with novel molecules and other tailored therapies. This syndicated approach has the potential to accelerate treatments for the 15,000 rare disorders while lowering the cost of drug development.

"This project really leverages what we already know as neuroscientists - that although these disorders have different genetic etiologies - there is considerable overlap in the disruptions to cellular biology. As such, I predict that we will find compounds which could be beneficial for several of our disorders participating in this project", said Anna Pfalzer, PhD, COMBINED Brain Chief Scientific Officer.

"Rare diseases are not rare, just very fragmented. This landmark collaboration with COMBINED Brain will demonstrate the potential to meaningfully address urgent patient needs by scaling rare disease therapeutics discovery to an unprecedented level," said Richard Novak, Ph.D., Unravel Co-Founder and CEO and Termeer Fellow.

About Unravel Biosciences
Unravel Biosciences is a therapeutics company founded on the principle that biological networks inform clinical outcomes. By using primary patient data with AI systems biology computation, rapid in vivo screening, and clinical validation of discovered targets, Unravel enables drug development with unprecedented efficiency. Unravel leverages its proprietary BioNAV™ platform combining target and drug prediction with patient stratification by efficacy and toxicity response to find treatments for complex diseases. Unravel's platform has led to two clinical trials starting in 2025. Unravel's platform developed RVL002, a first-in-class new small molecule targeting mitochondrial metabolism, and RVL027 and RVL068, molecules targeting two novel mechanisms 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.

About COMBINED Brain
The Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders (COMBINED Brain) is a non-profit consortium representing more than 110 rare neurological disorders. Their mission is to expedite meaningful treatments for individuals diagnosed with these disorders by pooling efforts across clinicians, scientists, advocates, and industry. For more information, visit www.combinedbrain.org.