ArpahThere are more than 10,000 known rare diseases and only a few hundred have safe, effective treatments. ARPA-H’s latest project aims to change that and deliver hope and improved outcomes to millions of Americans.

Introducing MATRIX. Short for ML/AI-Aided Therapeutic Repurposing in eXtended uses, MATRIX intends to build a machine learning platform to rapidly pinpoint and validate existing medications to treat diseases that have no therapies.

• The platform will be open-sourced and include an interactive heatmap displaying predicted efficacy scores for approved drugs against other diseases.

ARPA-H is allocating up to $48 million to the non-profit Every Cure in support of this research, with a potential for additional funds to validate the most promising drug-disease matches in clinical trials.

• The announcement was made by ARPA-H Director Renee Wegrzyn during a White House Rare Disease Forum with remarks from Every Cure Co-Founder Dr. David Fajgenbaum.

“Rather than the current, one step-at-a-time drug discovery process, we have an opportunity to use artificial intelligence to rapidly understand how already approved drugs could be effective against other diseases,” said Wegrzyn at the event. “It’s exciting to consider the potential of a systematic approach to identifying and validating the top repurposing candidates across all drug-disease combinations.”

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