Jaguar Launches with a Pre-clinical Pipeline of AAV9-Based Gene Therapies

After remaining in stealth mode in the past year, Jaguar Gene Therapy and its ciliopathy-focused subsidiary Axovia Therapeutics has launched itself with a Series A to develop adeno-associated virus serotype 9 (AAV9)-driven gene therapies.

This new venture is led by Jaguar’s former AveXis and Deerfield management teams, both of which provide access to a total of 18 established, crucial academic institutions for future Jaguar pipeline programs, such as the pre-clinical pipeline of AAV9-based gene therapies to fight against severe genetic diseases.

“Jaguar is uniquely positioned to accelerate breakthroughs in gene therapy for patients suffering from severe genetic diseases,” said Sean Nolan, Executive Chairman of Jaguar Gene Therapy. “We believe that the combination of our proven management team and gene therapy expertise coupled with the financial resources and access to academic institutions provided by Deerfield Management will facilitate our ability to identify critical clinical needs and address them by expediting the development of gene therapies from the bench to bedside as safely and quickly as possible.”

Leading Up to 2021

Novartis struck an $8.7 billion deal to buy AveXis in mid-2018 and gained control of gene therapy treatment of the rare neuromuscular disorder, spinal muscular atrophy (SMA), plus the resources AveXis constructed to support the lead candidate and discover future prospects.

AveXis’ SMA gene therapy demonstrated that its one-time shot outcompeted Biogen’s Spinraza in terms of convenience and efficacy. With that, Novartis evaluated that SMA gene therapy could yield billions of dollars in annual sales and became a leader in CAR-T and AAV9 gene therapies, two of the most promising fields in biotech.

Pre-clinical Pipeline of AAV9-Based Gene Therapies

The pre-clinical pipeline of AAV9-based gene therapies mainly emphasizes four genetic diseases: galactosemia, autism spectrum disorder, Type 1 diabetes, and Bardet-Biedl syndrome (BBS).

Currently underway, JAG101 aims to help patients dealing with galactosemia, affecting about 4,500 patients and an additional 17,000 individuals in the US. Galactosemia is an inborn error of carbohydrate metabolism, impairing the body’s ability to produce and process energy from galactose, a type of sugar in milk. Galactosemia leads to several consequences, including speech abnormalities, cataracts, liver failure, and kidney dysfunction. The current treatments place patients on a strict diet; unfortunately, such a standard of care still cannot prevent long-term complications.

JAG201 treats a specific genetic cause of autism spectrum disorder, victimizing 30,000 individuals a year in the US. Autism disorders trigger seizures, emotional or social interaction issues, as well as restricted and repetitive behaviors. Thus far, there is no available treatment for this genetic disorder.

JAG301 targets Type 1 diabetes which has the greatest number of sufferers worldwide than the three previously mentioned diseases. The prevalence is 9.5%, 15 per 100,000 people. Type 1 diabetes gives rise to a number of complications: blindness, heart disease, stroke, kidney damage, and much more. Type 1 diabetes is a metabolic, autoimmune disease, and its current treatment depends on lifelong intravenous injecting of insulin.

Lastly, AXV101 counteracts a subset of Bardet-Biedl syndrome (BBS), a life-threatening neurometabolic condition, and has influenced about 3,000 individuals in the US. Serious complications include progressive vision loss, obesity, learning disorders, and kidney disease. Sadly, there is no available treatment for BBS.

By Judy Ya-Hsuan Lin

Related Article: Sanofi Joins Hands with German Biotech to Develop Improved AAV Capsids for Gene Therapies

References

1. https://www.businesswire.com/news/home/20210225005204/en/Jaguar-Gene-Therapy-Launches-with-Mission-to-Accelerate-Breakthroughs-in-Gene-Therapy-for-Patients-Suffering-from-Severe-Genetic-Diseases?fbclid=IwAR3sTiU8lt6-Ds5xzWEaYChrL-UoX-ekmGatQIvUa7uMrAgiVyhb6EPgzpI

Author

Judy Ya-Hsuan Lin

Success is the ability to go from one failure to another with no loss of enthusiasm.