Eli Lilly and Profluent form USD 2.25b partnership on AI-designed recombinases for genetic meds

Emeryville, California-based Profluent and Indianapolis-based Eli Lilly and Company (NYSE: LLY) have announced a multi-program strategic research collaboration to design and commercialize custom site-specific recombinases for diseases with high unmet need. The collaboration applies Profluent’s AI foundation model platform to generate novel recombinase enzymes programmed to recognize precise genomic loci, with Lilly responsible for advancing selected candidates through in vivo research, preclinical development, clinical studies, and commercialization under an exclusive license. Specific disease indications and genomic targets have not been disclosed.

Profluent will receive an upfront payment alongside committed research and development funding, with amounts undisclosed. The company is eligible for up to USD 2.25 billion in development and commercial milestone payments, plus tiered royalties on net sales. No per-program milestone breakdown, royalty rate ranges, or geographic carve-outs were disclosed; the exclusive license to Lilly is implied to be global based on the language of the announcement.

Profluent’s platform uses large-scale generative AI models trained on what the company describes as the world’s largest curated recombinase dataset, alongside broad protein universe data, to design enzymes that can recognize user-specified DNA sequences. Unlike nuclease-based editing approaches such as CRISPR-Cas9, site-specific recombinases catalyze DNA recombination without inducing double-strand breaks, enabling insertion, deletion, or inversion of large DNA segments at targeted genomic locations. The ability to insert kilobase-scale payloads at precise sites addresses a longstanding limitation of first-generation editing tools, which have faced constraints around payload size and off-target activity driven by break-and-repair mechanisms.

Under the collaboration’s work-share structure, Profluent will apply its AI models to design and optimize recombinases across multiple genomic targets. Lilly will select candidates and lead all downstream development and commercialization activity. No co-promotion, co-development option, or profit-sharing alternative to the royalty structure was described.

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Lilly has been assembling a portfolio of genetic medicine capabilities through external partnerships. The company’s gene therapy unit, Akouos, has an active program in OTOF-associated hearing loss in clinical development. In 2025, Lilly entered a separate licensing agreement with Rznomics covering RNA-editing therapeutics for sensorineural hearing loss. In 2026, Lilly also announced a collaboration with Seamless Therapeutics to develop programmable recombinase-based therapeutics specifically for hearing loss indications, representing a parallel recombinase-focused effort within the same modality as the Profluent collaboration. The Profluent deal’s undisclosed target set could overlap with or extend beyond that disease area, though no indication has been confirmed publicly.

The broader gene-editing field has reached a regulatory inflection point that increases the strategic rationale for platform-level investments. CRISPR-based therapies for sickle cell disease received regulatory approval in 2023, establishing inherited disease as a validated commercial and regulatory category for gene editing. In the hearing-loss space specifically, Regeneron’s Otarmeni (lunsotogene parvec-cwha) was reported in 2026 as the first approved gene therapy for OTOF-associated severe-to-profound hearing loss, demonstrating that genetic hearing disorders can advance through the full regulatory pathway. These precedents reduce platform-level uncertainty for recombinase-based approaches targeting genetically defined conditions.


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