The UC Berkeley Molecular Therapeutics Initiative (MTI) announced a new collaboration agreement with BioMarin Pharmaceutical Inc. aimed at accelerating the discovery and development of innovative therapies for rare genetic conditions. The partnership brings together UC Berkeley’s strengths in fundamental disease biology, novel therapeutic technologies, and next-generation drug discovery, with BioMarin’s deep expertise in translating science into medicines for people living with rare genetic conditions.
The initial phase of the collaboration will support specific joint research projects between BioMarin scientists and UC Berkeley investigators in the laboratories of Margaux Pinney, Roberto Zoncu, and Daniel Nomura. These projects will leverage UC Berkeley MTI’s drug discovery infrastructure and translational capabilities, with the opportunity to expand the collaboration over time to additional investigators across the MTI.
“This collaboration reflects MTI’s core mission: to connect cutting-edge academic science with real-world drug development,” said Daniel Nomura, co-director of the UC Berkeley Molecular Therapeutics Initiative. “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.”
This collaboration reflects MTI’s core mission: to connect cutting-edge academic science with real-world drug development
Dan Nomura
MTI faculty Co-director
Roberto Zoncu, co-director of MTI, emphasized the scientific depth of the partnership. “Rare genetic diseases often illuminate fundamental biology in powerful ways,” Zoncu said. “This collaboration creates a unique environment where basic mechanistic insights can be rapidly translated into therapeutic strategies, supported by BioMarin’s exceptional translational and clinical expertise.”
Julia Schaletzky, executive director of the UC Berkeley Molecular Therapeutics Initiative, highlighted the broader platform value of the partnership. “MTI was built to enable exactly this kind of collaboration—one that combines investigator-driven science, shared drug discovery capabilities, and strong industry partnership,” Schaletzky said. “We see this agreement as a foundation for long-term, scalable collaboration across the Berkeley ecosystem.”
“BioMarin has a long and dedicated history of partnering with leading academic institutions like UC Berkeley to advance the understanding of disease biology,” said Kevin Eggan, Chief Scientific Officer at BioMarin. “This collaboration with the UC Berkeley Molecular Therapeutics Initiative will enable us to engage directly with world-class investigators and emerging technologies that can open new paths for helping people living with rare and genetic conditions.”
The collaboration reinforces UC Berkeley MTI’s role as a hub for translational research and drug discovery, bridging academic innovation and industry development to address diseases with significant unmet medical need.
This collaboration with the UC Berkeley Molecular Therapeutics Initiative will enable us to engage directly with world-class investigators and emerging technologies that can open new paths for helping people living with rare and genetic conditions.
Kevin Eggan
Chief Scientific Officer, Head of Research and Early Development, BioMarin
