Jaguar Gene Therapy is advancing an initial pipeline of three AAV-based, preclinical gene therapy programs targeting Type 1 galactosemia (JAG101), SHANK3-related neurodevelopmental disorders including Phelan-McDermid syndrome (JAG201), and Type 1 diabetes (JAG301). The company emphasizes first-in-class CMC (Chemistry, Manufacturing and Controls) capabilities and commercially scalable manufacturing processes for AAV therapeutics. Jaguar says it will use the proceeds from the new investments, together with existing cash resources, to expand CMC capabilities and to advance IND-enabling activities across its preclinical pipeline. The team highlights prior experience that includes overseeing development and launch of one of the first FDA‑approved gene therapies. Jaguar positions JAG301 as a cure-oriented program aiming to restore endogenous insulin production via PAX4-mediated transdifferentiation of alpha cells. The company continues to evaluate opportunities to expand its investigational gene therapy pipeline. Jaguar Gene Therapy, based in Lake Forest, Ill., develops AAV9-based gene therapies for patients with severe genetic diseases. Led by former AveXis leadership, the company is advancing a pre-clinical pipeline including JAG101 for galactosemia, JAG201 for a genetic cause of autism spectrum disorder, and JAG301 for Type 1 diabetes. JAG101 targets galactosemia, a condition affecting an estimated 4,500 patients in the U.S. in its most severe form and an additional ~17,000 with less severe disease; current standard of care is dietary and often insufficient. JAG201 targets an estimated 30,000 U.S. patients with a genetically caused autism spectrum disorder for which no treatments exist, while JAG301 is evaluating proof-of-concept data to define clinically relevant newly diagnosed Type 1 diabetes patients. A majority-owned subsidiary, Axovia Therapeutics, is advancing AXV101 for BBS1, a subset of Bardet-Biedl syndrome. Jaguar emphasizes development, manufacturing and commercialization capabilities and intends to use recent financing and existing cash to advance its initial pre-clinical programs.

Total raised

$139M

Funding rounds

2

Latest round

Series B

Latest activity

Apr 2021