Addressing genetic disorders head-on
Our priority is to help patients with significant unmet needs and address them by expediting the development of gene therapies from bench to bedside as safely and quickly as possible. We created our initial pre-clinical pipeline by identifying diseases where a one-time gene therapy solution can have a major impact on the trajectory of a person’s life.
Initial pre-clinical pipeline
Our initial pre-clinical pipeline of AAV9-based gene therapy programs targets diseases with significant unmet need.
a gene therapy treatment in development for galactosemia, a metabolic condition that impairs the body’s ability to process and produce energy from galactose, leading to long-term complications
a gene therapy treatment in development for a specific genetic cause of autism spectrum disorder
a gene therapy treatment in development for Type 1 diabetes, a metabolic autoimmune disease that currently requires lifelong insulin injection dependency
a gene therapy treatment for BBS1, a subset of Bardet-Biedl syndrome (BBS), a life-threatening neurometabolic condition. This program is being developed by Axovia Therapeutics, a majority-owned subsidiary of Jaguar Gene Therapy
- Target Identification
BBS1, a subset of Bardet-Biedl syndrome (BBS)
This program is being developed by Axovia Therapeutics, a majority-owned subsidiary of Jaguar Gene Therapy, that is focused on creating transformative therapies for ciliopathies. To learn more about Axovia Therapeutics, visit their website.
We are game changers
The Jaguar Gene Therapy team is led by high-performing executives with proven experience in bringing gene therapies to market.