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 AAV-based gene therapy programs targets diseases with significant unmet need.
an investigational gene therapy currently in preclinical development that aims to deliver a gene replacement solution to address the root cause of Type 1 galactosemia by delivering the functional GALT gene via the AAV9 vector
an investigational gene therapy currently in preclinical development that aims to deliver appropriate SHANK3 genetic function via the AAV9 vector to treat the root cause of the disease and rescue behavioral abnormalities
an investigational AAV-based gene therapy currently in preclinical development that works to address the root cause of the disease by producing functional beta cells using the PAX4 gene to convert alpha cells to beta cells, a process called transdifferentiation
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.