COMBINED Brain and Unravel Biosciences Enter Rare Neuro Drug Discovery Collaboration

Rare Daily Staff

AI-enabled drug developer Unravel Biosciences and the non-profit consortium COMBINED Brain said they have initiated 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.

The Harvard University spin-out Unravel will utilize its proprietary BioNAV drug discovery platform and COMBINED Brain’s 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.

The collaboration said it 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,” said Terry Jo Bichell, CEO of COMBINED Brain CEO. “This project is a way to identify drugs which we could pilot in clinical trials across multiple rare pediatric diseases at the same time.”

The resulting datasets and drug candidates will accelerate the efforts of member patient advocacy groups to identify promising drugs. The overall goal is 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 all 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,” said Anna Pfalzer, chief scientific officer of COMBINED Brain. “As such, I predict that we will find compounds which could be beneficial for several of our disorders participating in this project.”

Photo: Terry Jo Bichell, CEO of COMBINED Brain