RARE Daily

Exsilio Therapeutics Launches with $82 Million to Develop Redosable Genomic Medicines

June 25, 2024

Rare Daily Staff

Exsilio Therapeutics emerged from stealth with $82 million in series A financing to advance its genomic medicines based on naturally occurring, programmable genetic elements that can precisely insert new genes into a cell through mRNA intermediates.

Novartis Venture Fund and Delos Capital led the financing with participation from OrbiMed, Insight Partners, J.P. Morgan Life Sciences Private Capital, CRISPR Therapeutics, Innovation Endeavors, Invus, Arc Ventures, and Deep Insight. OrbiMed had provided seed funding to Exsilio.

Exsilio has built a platform that combines predictive in silico modeling and wet lab-based experimentation to discover and engineer elements for integration of therapeutic genes into safe harbor sites. Because Exsilio’s medicines are encoded in mRNA, they can be delivered using existing lipid nanoparticle platforms that are safe, efficient, scalable, and cost-effective, and can be redosed and titrated with curative intent.

“Exsilio’s approach leverages the advantages of mRNA and goes a step further by encoding genes that integrate permanently, offering the possibility of curative rather than transient effects,” said Tal Zaks, Exsilio’s chairman and interim CEO. “The ability to insert whole genes with a repeatable and titratable approach should allow us to treat genetic diseases irrespective of the patient’s individual mutation.”

Photo: Tal Zaks, Exsilio’s chairman and interim CEO

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