Exsilio Therapeutics ("Exsilio"), a cutting-edge biotechnology company specializing in genomic medicines, announced its emergence from stealth mode today, securing $82 million in Series A financing. Exsilio plans to use the proceeds from this financing to advance its platform for developing genomic medicines based on naturally occurring, programmable genetic elements. 

These elements are capable of precisely inserting new genes into cells via mRNA intermediates. Exsilio’s interdisciplinary team has developed a platform that integrates predictive in silico modeling with wet lab-based experimentation to identify and engineer these genetic elements for safe and effective therapeutic gene integration.

The company's genomic medicines, encoded in mRNA, can be delivered using existing lipid nanoparticle (LNP) platforms. These platforms are known for their safety, efficiency, scalability, and cost-effectiveness. Moreover, Exsilio's approach allows for redosing and titration with the aim of achieving curative outcomes.

"mRNA-based medicines allow for a software-like approach to creating new medicines," said Tal Zaks, M.D., Ph.D., who serves as Exsilio's Chairman and Interim Chief Executive Officer. "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. 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. This financing will help us advance our genomic medicines and select promising lead candidates so that we can bring much-needed new options to patients."

"We were captivated by Exsilio's genomic medicines approach that stands to enable large-gene integration in a safe and redosable manner," said Aaron Nelson, Managing Director at Novartis Venture Fund and Exsilio Board Member. "Through this significant investment, Exsilio will be able to select and advance promising candidates for difficult-to-treat diseases."

"We believe that the vision of safely and durably integrating therapeutic genes into a patient's genome requires using RNA-based payloads that can leverage clinically validated non-viral gene delivery technologies," said Henry Chen, Managing Partner of Delos Capital and Exsilio Board Member. "Exsilio is bringing together a singular group of people to help establish this new pillar of genomic medicine."

Exsilio's innovative approach promises to pave the way for new treatments for a broad range of genetic diseases, bringing hope to patients who have long awaited effective therapies.

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