BioMarin and Deep Genomics to Collaborate on AI Drug Discovery for Rare Diseases

Rare Daily Staff

BioMarin Pharmaceutical and Deep Genomics have entered into a preclinical collaboration that will use Deep Genomics’ artificial intelligence drug discovery platform to identify oligonucleotide drug candidates in four rare disease indications with high unmet need.  

Deep Genomics will receive an undisclosed upfront payment and is eligible to receive development milestones as a part of the collaboration.  BioMarin will receive an exclusive option to obtain Deep Genomics’ rights to each program for development and commercialization. The companies did not disclose financial terms.

“We believe the combination of Deep Genomics’ experience in using artificial intelligence to creatively modulate targets coupled with our proven track record in developing transformational medicines for patients with rare diseases will speed BioMarin’s trajectory into new biological frontiers,” said Lon Cardon, chief scientific strategy officer and senior vice president at BioMarin.

Deep Genomics uses AI to power every stage of drug development, from identifying therapeutic targets that were previously dismissed as being undruggable, to designing novel therapeutic candidates, to designing animal models.

Under their collaboration, Deep Genomics will use its AI Workbench to identify and validate target mechanisms and lead candidates, and BioMarin will advance them into preclinical and clinical development. The AI Workbench enables rapid exploration of novel targetable mechanisms and therapeutic candidates.  It combines deep learning, automation, advanced biomedical knowledge and massive amounts of in vitro and in vivo data to accurately identify targetable molecular mechanisms and guide the discovery and development of oligonucleotide therapies.

“Our second generation AI Workbench continues to unlock a rapidly growing number of therapeutic opportunities for patients with genetically defined disorders,” said Brendan Frey, founder and CEO of Deep Genomics. “BioMarin is an industry leader in developing transformational therapies for patients with rare diseases, and we look forward to working with them to expand their clinical pipeline.”

Photo: Lon Cardon, chief scientific strategy officer and senior vice president at BioMarin.