UC Berkeley Partners with BioMarin to Accelerate Rare Disease Research

The UC Berkeley Molecular Therapeutics Initiative (MTI) has entered into a new research collaboration with BioMarin Pharmaceutical to advance the discovery and development of treatments for rare genetic diseases.

The partnership combines UC Berkeley’s expertise in disease biology and novel therapeutic technologies with BioMarin’s experience translating research into approved medicines. Under the agreement, BioMarin scientists will work with UC Berkeley researchers in the laboratories of Margaux Pinney, Roberto Zoncu, and Daniel Nomura to pursue joint projects aimed at uncovering disease mechanisms and developing potential therapies.

The partnership is part of a broader effort to create a scalable, long-term platform for academic–industry collaboration across Berkeley’s research ecosystem.

“This collaboration reflects MTI’s core mission: to connect cutting-edge academic science with real-world drug development,” said Nomura, MTI co-director. “By pairing UC Berkeley’s strengths in uncovering disease mechanisms and inventing new therapeutic modalities with BioMarin’s leadership in developing medicines for rare genetic conditions, we can accelerate the path from discovery to impact.”

MTI serves as a hub for translational research and drug discovery at UC Berkeley, linking academic innovation with industry development to address diseases with significant unmet medical needs.

Photos: Daniel Nomura, co-director of MTI