Addressing genetic disorders head-on

Our first priority is helping patients with high unmet medical need by advancing transformative AAV-based gene therapy treatments. There’s not a moment to waste. That’s why we set clear objectives, rapidly find targets, identify solutions — and deliver results.

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Current Therapeutic Areas of Focus

We are building a pipeline of gene therapies starting with these therapeutic areas.

Neurodevelopmental

Caused by disruption of the brain or central nervous system and characterized by abnormal brain function.

Neurometabolic

Affects the body’s ability to perform standard metabolic processes and functions within the brain or central nervous system

Metabolic

Affects the body’s ability to perform standard metabolic processes

  • Description
  • Target Identification
  • Product Optimization
  • Preclinical
  • Clinical

Bardet-Biedl Syndrome (BBS)

With operational support from Jaguar Gene Therapy, Axovia Therapeutics is accelerating the development of AAV-based gene therapies for patients with Bardet-Biedl Syndrome (BBS). 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.