Profluent and BioMarin Pharmaceutical have entered a collaboration focused on designing and optimizing novel enzyme therapies for BioMarin’s pipeline, combining artificial intelligence-based protein design with the pharmaceutical company’s experience developing enzyme replacement therapies for genetically defined conditions.
Under the agreement, Profluent will use its foundational AI models to generate potential drug candidates. BioMarin will then screen the resulting designs, with the aim of identifying candidates for further evaluation and potentially advancing selected molecules into preclinical testing.
Therapeutic enzyme development can require extensive protein engineering and screening to identify molecules with appropriate characteristics. Profluent plans to apply its proprietary protein language models to accelerate this process, enabling BioMarin to explore a broader range of protein designs and identify candidates with targeted activity and stability profiles.
The models use the Profluent Protein Atlas, which contains more than 115 billion unique protein sequences. The companies intend to combine this computational approach with BioMarin’s experience in enzyme therapies to support the development of potential treatments for patients with significant unmet needs.
“Profluent’s AI models represent the kind of external innovation that can complement our own capabilities and help us move promising science forward faster,” said Pooja Agarwal, Ph.D., Vice President and Head of Metabolic Conditions Research at BioMarin. “By combining Profluent’s approach to de novo protein design and optimization with BioMarin’s decades of expertise in enzyme therapies, we have an opportunity to identify promising candidates more efficiently for people with significant unmet need.”
Profluent CEO Ali Madani also highlighted the companies’ complementary capabilities.
"BioMarin has one of the most credible track records in enzyme therapies, spending decades successfully advancing drugs through the clinic to improve the lives of patients," said Madani. "We're excited to accelerate BioMarin's drug discovery pipeline and unlock new medicines by combining the strengths of our latest foundation models for protein design with their deep track record of getting therapies to patients."
The collaboration reflects the companies’ plans to apply AI-based protein design directly to BioMarin’s drug discovery pipeline, with BioMarin retaining responsibility for evaluating generated candidates and advancing the most promising designs toward preclinical development.
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