BioMarin partners with Profluent to develop AI-designed enzyme therapies in USA

USA—BioMarin Pharmaceutical has entered a research collaboration with Profluent to design and optimize novel enzyme therapies for its drug development pipeline, using artificial intelligence (AI) to identify potential therapeutic candidates.

Under the agreement, Profluent will use its protein language models to generate candidate enzymes, while BioMarin will screen and evaluate the resulting designs for target activity and stability.

The pharmaceutical company will advance selected candidates into preclinical testing.

AI models to support enzyme discovery

Developing therapeutic enzymes requires extensive protein engineering and laboratory screening to identify molecules with the properties needed for a particular treatment.

AI-based protein design offers researchers another way to explore potential candidates before committing resources to experimental testing.

Profluent will conduct the computational design work using its Profluent Protein Atlas, a dataset containing more than 115 billion unique protein sequences.

The company describes the resource as the world’s largest protein sequence repository.

The platform will support generating and optimising candidate enzymes for BioMarin’s research programmes.

By computationally assessing alternative protein designs, BioMarin aims to identify promising molecules for further laboratory evaluation.

BioMarin’s vice-president and head of metabolic conditions research, Pooja Agarwal, said the collaboration would bring external AI capabilities into the company’s research programmes.

She said combining Profluent’s approach to designing and optimising proteins from scratch with BioMarin’s experience in enzyme therapies could help researchers identify candidates more efficiently for diseases with significant unmet medical needs.

Companies combine computational design and therapeutic development

Profluent chief executive officer Ali Madani said BioMarin’s experience developing enzyme therapies made it a suitable partner for applying the company’s protein design technology to drug discovery.

He said the collaboration would combine Profluent’s latest protein-design foundation models with BioMarin’s experience advancing treatments through clinical development and into patient care.

The agreement places computational protein engineering within BioMarin’s broader drug discovery activities.

The partnership will focus on generating potential therapeutic molecules, with laboratory screening and subsequent preclinical evaluation determining which designs progress through development.

For biotechnology companies, this approach offers a way to expand the range of proteins considered during early research.

However, computationally generated candidates must still demonstrate the required biological activity, stability and safety through experimental studies before they can advance towards clinical testing.

Profluent expands its AI-driven biotechnology work

Founded in 2022 and based in Emeryville, California, Profluent develops biological foundation models for applications in medicine, agriculture and other fields.

Its technology uses large protein sequence datasets to support the design and optimisation of biological molecules.

The company’s investors include AIX Ventures, Air Street Capital, Bezos Expeditions, Altimeter Capital, Insight Partners, Convergent Ventures and Spark Capital.

The collaboration builds on BioMarin’s focus on enzyme-based treatments for genetic and metabolic disorders, where therapeutic proteins can address specific biological deficiencies.

The research agreement gives the company access to computational design capabilities while retaining a role in evaluating candidates for its development pipeline.

In April 2026, BioMarin completed its acquisition of Amicus Therapeutics in an all-cash transaction valued at nearly US$4.8 billion in total equity value.

The acquisition expanded BioMarin’s rare-disease portfolio, while its agreement with Profluent adds an AI-enabled research collaboration to its drug discovery efforts.

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