Jaguar Gene Therapy launches, targeting rare genetic diseases

By The Science Advisory Board staff writers

February 25, 2021 -- Jaguar Gene Therapy has launched after being in stealth mode with the goal of accelerating the development, manufacturing, and commercialization of novel gene therapy treatments for patients with severe genetic diseases.

The company, which has been operating in stealth mode for the past year, is focused on advancing its preclinical pipeline of gene therapies based on adeno-associated virus 9 (AAV9). The company is targeting diseases with significant unmet needs such as galactosemia, genetic causes of autism spectrum disorder, type 1 diabetes, and Bardet-Biedl syndrome (BBS).

AXV101, a candidate for BBS1, a subset of Bardet-Biedl syndrome, is being developed by Axovia Therapeutics, a majority-owned subsidiary of Jaguar Gene Therapy.

The new venture was created in collaboration with Deerfield Management, which funded the series A financing and helped establish academic collaborations for pipeline projects. Through Deerfield, Jaguar has access to an existing network of 18 key academic institutions.

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