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Profluent, a biotechnology company focused on computational protein engineering, and Ensoma, an in vivo cellular engineering firm developing one-time genomic therapies, have announced a research collaboration to evaluate the application of AI-designed deaminase enzymes within Ensoma’s hematopoietic stem cell (HSC) gene-editing platform. The partnership aims to integrate Profluent’s de novo–designed deaminases with Ensoma’s proprietary gene-editing scaffolds and helper-dependent adenovirus delivery systems to develop and assess novel base editor constructs for in vivo HSC modification. Data generated through the collaboration are intended to guide downstream translational development decisions.
Gene editing has profound potential to transform biomedical research and how we treat disease. Generative AI lets us sculpt enzymes to the biological context that matters. We are thrilled to be working with Ensoma to support the future of their incredible platform and the potential of our editor designs for in vivo HSC settings, where precision and durability count.
Ali Madani, Ph.D., Founder and CEO of Profluent
HSCs possess both long-term self-renewal capacity and multilineage differentiation potential, making them a central target for durable therapeutic interventions in hematologic and immune disorders. Therapeutic editing of HSCs directly in vivo offers an alternative to ex vivo manipulation and transplantation approaches, potentially enabling sustained production of genetically modified blood and immune cells after a single treatment. Ensoma has previously reported clinical entry with an in vivo HSC insertion therapy platform.
AI protein design is a potentially powerful enabler to our vision for one-time treatments addressing a wide range of serious diseases, including many difficult-to-treat genetic conditions. Profluent’s platform for precision design of gene editors is very exciting. Their technology offers more opportunities to potentially deliver the next wave of in vivo gene editing.
Jim Burns, CEO of Ensoma
The collaboration will focus on characterizing the activity, specificity, and editing efficiency of AI-designed base editor systems in the context of HSC targeting. Advances in computational protein design have enabled systematic optimization of deaminase enzymes, potentially improving the balance between catalytic activity and editing precision.
Beyond the Ensoma collaboration, Profluent has applied its foundation model platform to multiple gene-editing research programs since the release of its OpenCRISPR-1 system, which supports community access to AI-designed editing tools. Additional partnerships have been announced with Revvity to develop enhanced base-editing systems and with the Rett Syndrome Research Trust to design mutation-specific gene editors for rare neurodegenerative disorders.