Profluent Bio Partners with Ensoma for AI-Designed Base Editors in Stem Cell Therapies

Profluent, a frontrunner in AI for protein design, has announced a strategic partnership with Ensoma, a company focused on in vivo cellular engineering aimed at enhancing medical therapies through one-time treatments. This collaboration will utilize Profluent’s AI-designed deaminases alongside Ensoma’s gene editing scaffolds and helper-dependent adenovirus (HDAd) delivery systems. Together, they plan to design, develop, and assess AI-designed base editors specifically for in vivo hematopoietic stem cell (HSC) therapies, which will guide future translational decisions.

“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," stated Jim Burns, CEO of Ensoma.

Hematopoietic stem cells are unique for their ability to self-renew and differentiate into various blood and immune cells. Editing these cells in vivo can potentially provide a sustainable source of engineered cells aimed at treating specific hematologic and immune disorders. Ensoma has already made strides in HSC engineering, being the first to introduce an in vivo therapeutic for gene insertion into HSCs. Profluent's advancements in AI-designed base editors may lead to innovative in vivo therapies that ensure both efficacy and precision.

“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,” remarked Ali Madani, Ph.D., Founder and CEO of Profluent. “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.”

“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,” added Jim Burns. “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.”

Since the launch of OpenCRISPR-1TM, Profluent has been applying its advanced AI capabilities to a variety of gene editing applications. The company has recently formed partnerships with Revvity to create enhanced base editing systems and with the Rett Syndrome Research Trust to develop custom gene editors targeting mutations responsible for the rare neurodegenerative disorder.