Profluent and Eli Lilly partner to develop AI-designed recombinases for precise, large-scale DNA editing, targeting genetic diseases beyond current gene editing approaches.
Written By: Nikita Jha, BPharm
Reviewed By: Pharmacally Editorial Team
Profluent has announced a multi-program research collaboration with Eli Lilly and Company focused on developing custom site-specific recombinases for genetic diseases with unmet medical needs.
The partnership combines Profluent’s AI-driven protein design platform with Lilly’s expertise in genetic medicines, aiming to enable more precise and scalable DNA editing approaches beyond current gene editing technologies.
Many genetic disorders involve significant variation across patients, making them difficult to address with conventional tools such as knock-out or base editing. One potential solution is large-scale, site-specific DNA integration, including the insertion of long DNA sequences or entire genes at defined genomic locations. However, this remains technically challenging and not yet widely scalable.
Profluent is using AI-trained models to design and optimize recombinases enzymes capable of targeted DNA rearrangement expanding beyond naturally occurring systems. Under the agreement, the company will develop and refine recombinases across multiple targets. Lilly will receive exclusive rights to advance selected candidates through in vivo research, preclinical development, clinical trials, and potential commercialization.
“Kilobase-scale DNA editing remains a holy grail in genetic medicine,” said Ali Madani, co-founder and CEO of Profluent. “Our work with Lilly is aimed at unlocking these therapeutics.”
Hilary Eaton, Chief Business Officer at Profluent, added that existing editing approaches leave major categories of disease unaddressed, and that advances in large-scale DNA editing could expand the scope of treatable conditions.
Profluent’s models are trained on large-scale protein datasets, including recombinase sequences, with the goal of building a programmable platform applicable to both rare and common diseases.
Under the terms of the agreement, Profluent will receive an upfront payment, research funding, and up to $2.25 billion in milestone payments, along with tiered royalties on net sales. Additional financial details were not disclosed.
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About the Writer
Nikita Jha, BPharm a pharmacy graduate specializing in medical writing, with a strong ability to interpret complex medical and regulatory information and translate it into clear, accurate, and evidence-based healthcare content. Known for her attention to detail and precision, she focuses on delivering high-quality scientific communication that supports drug safety and informed decision-making.
