Series A co-led by Novartis Venture Fund and Delos Capital, with participation from OrbiMed, Insight Partners, J.P. Morgan Life Sciences Private Capital, CRISPR Therapeutics, Innovation Endeavors, Invus, Arc Ventures, and Deep Insight

Company is developing a new class of genomic medicines based on naturally occurring genetic elements that may be delivered using mRNA and lipid nanoparticles to precisely and durably insert entire genes into safe harbor sites in the human genome

Company is focused on advancing medicines that can be redosed and titrated with curative intent for patients suffering from genetic diseases, cancer, and autoimmune conditions

BOSTON, June 25, 2024 /PRNewswire/ -- Exsilio Therapeutics ("Exsilio"), a biotechnology company developing genomic medicines for a broad range of diseases, announced today its emergence from stealth with $82 million in Series A financing. The funding was co-led by Novartis Venture Fund and Delos Capital, with participation from OrbiMed, Insight Partners, J.P. Morgan Life Sciences Private Capital, CRISPR Therapeutics, Innovation Endeavors, Invus, Arc Ventures, and Deep Insight. Exsilio was seed-funded by OrbiMed.

Exsilio will use proceeds from the 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. Exsilio's interdisciplinary team has built a platform that combines predictive in silico modeling and wet lab-based experimentation to discover and engineer such 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 (LNP) platforms that are safe, efficient, scalable, and cost-effective, and can be redosed and titrated with curative intent.

"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."

About Exsilio Therapeutics
Exsilio Therapeutics is developing genomic medicines that can be delivered in lipid nanoparticles to durably and safely treat a broad range of diseases, including genetic diseases, cancer, and autoimmune conditions. Exsilio's technology is based on naturally occurring genetic elements that can precisely insert new genes into a cell through RNA intermediate templates. Exsilio uses predictive in silico modeling and wet lab-based experimentation to engineer such elements for precise insertion of therapeutic genes into safe harbor sites of disease-relevant cells. Because Exsilio's medicines are encoded in mRNA, they can be delivered using validated, non-viral delivery platforms that are safe, efficient, scalable, and cost-effective, and that allow redosing to effect with a curative intent. For more information, follow us on LinkedIn.

SOURCE Exsilio