Eli Lilly and Profluent Announce $2.25B Strategic Collaboration to Develop AI-Designed Recombinase-Based Gene Editing Therapies

On April 28, Profluent Bio, Inc., a frontier artificial intelligence company that pioneered large-scale foundational models for protein design,ProfluentAnnounced a multi-project strategic research collaboration with Eli Lilly to develop and commercialize custom site-specific recombinases to address significant unmet needs.Disease.Profluent claims to be the first company to prove that large language models can generate functional proteins, while referring to its atlas of over 115 billion unique proteins as "the world's largest protein data resource."Source”。Bezos Expeditions, led by Jeff Bezos, co-led the investment in Profluent last November.$106 million financing round,Bringing the total funding to $150 million to accelerate the design of genome editors, antibodies, and enzymes for real-world impact.AI Protein Design Company Raises $35 Million in Financing, Revolutionizing Gene Editing Field）

The focus of this collaboration is to achieve large-scale, precise DNA editing capabilities, which remain unattainable with traditional gene editing systems. By combining Profluent's cutting-edge artificial intelligence models with Eli Lilly and Company's clinical and development expertise in genetic medicines, the two companies aim to expand the scope and scalability of programmable gene editing therapies.

Many genetic diseases are caused by a variety of different mutations within the patient population rather than a single mutation. This heterogeneity makes it difficult to develop targeted therapies that are applicable to all patients. Large-scale DNA editing (the ability to insert long DNA fragments, sometimes entire genes, at precise locations in the genome) could address this challenge but remains one of the most significant unsolved problems in genetic medicine.

Traditional methods rely on finding natural enzymes that happen to work at the target site. Profluent is taking a different approach: using artificial intelligence to create designed recombinases — custom enzymes programmed to target precise locations in the genome.

According to the agreement,ProfluentProfluent will apply its artificial intelligence model to design and optimize site-specific recombinases for multiple genomic targets. Eli Lilly and Company will receive an exclusive license to advance selected recombinases through in vivo studies, preclinical development, clinical research, and commercialization.

In addition to the committed R&D funds,ProfluentWill also receive an advance payment. Overall,ProfluentEligible to receive up to $2.25 billion in development and commercial milestone payments, as well as tiered royalties on net sales. Further details of the agreement have not been disclosed.

Profluent Co-Founder and Chief ExecutiveOfficialAli Madani stated:"Editing DNA on the kilobase scale remains the holy grail of genetic medicine. Our collaboration with Eli Lilly aims to unlock treatments once thought impossible. We believe only artificial intelligence can create the designed recombinases needed to precisely target any location in the genome. This partnership with Lilly demonstrates that our platform is ready to build the toolkit necessary for expanding genetic medicines. We see strong demand from the pharmaceutical industry, and this collaboration validates our approach to solving previously intractable problems."

Profluent Chief Business Officer Hilary Eaton stated:"Standard knockout and base editing methods make entire categories of diseases unreachable. Kilobase-scale DNA editing is how we reach them, and Profluent's generative models were built to address this issue, based on the world's largest protein dataset, including the most comprehensive database of natural recombinases. Our goal is to create a fully programmable platform applicable to both rare and common diseases, and we believe that Eli Lilly and Company is the ideal partner to bring these tools to the patients who need them most."