RARE Daily

Former AveXis Executives Launch Jaguar Gene Therapy to Target Rare Diseases

February 25, 2021

Rare Daily Staff

Former executives of gene therapy pioneer AveXis publicly launched Jaguar Gene Therapy with a mission to accelerate the development, manufacturing and commercialization of novel gene therapy treatments for patients suffering from severe genetic diseases.

Launched one year after founding, Jaguar was created in collaboration with investment firm Deerfield Management, which funded the series A financing and provides access to established academic institutions for future pipeline programs.

“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, and former AveXis CEO. “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 bench to bedside as safely and quickly as possible.”

Jaguar’s initial preclinical pipeline utilizes the proven and well-characterized AAV9 vector to target diseases with significant unmet need, including galactosemia, genetic causes of autism spectrum disorder, Type 1 diabetes and Bardet-Biedl syndrome.

JAG101 is a gene therapy treatment in development for galactosemia, a metabolic condition that in its most severe form affects an estimated 4,500 patients in the United States. An additional estimated 17,000 individuals in the United States have a less severe form of the disease yet still suffer from long-term effects. Galactosemia is diagnosed within months of birth and is caused by an inborn error of carbohydrate metabolism, which impairs the body’s ability to process and produce energy from galactose, one of the sugars in breast milk and formula. Consequences of the disease can include cataracts, liver failure, kidney dysfunction and brain damage (speech abnormalities). Because of its severity, galactosemia has already been added as part of newborn screening in the United States and in other global markets. The current standard of care is a strict diet that has modest effects in some patients but is often not sufficient to prevent long-term complications.

JAG201 is a gene therapy treatment in development for a specific genetic cause of autism spectrum disorder. Hallmarks of autism disorders include seizures, emotional/social interaction issues, and restricted and repetitive behaviors that can persist and interfere with everyday life. There are currently no treatment options available for the estimated 30,000 patients in the United States with the genetically caused autism spectrum disorder that Jaguar is targeting.

JAG301 is a gene therapy treatment in development for Type 1 diabetes, a metabolic autoimmune disease that currently requires lifelong insulin injection dependency. Serious complications from Type 1 diabetes can include frequent hospitalizations, blindness, heart disease, stroke, kidney damage and nerve damage. Jaguar is currently evaluating proof- of-concept data to best determine the appropriate clinically relevant patients within the newly diagnosed population.

AXV101 is a gene therapy treatment for BBS1, a subset of Bardet-Biedl syndrome (BBS). A life-threatening neurometabolic condition, BBS affects approximately 3,000 patients in the United States. It causes progressive vision loss, severe obesity, learning disorders and kidney disease. Currently, there is no effective treatment available for BBS patients. Axovia Therapeutics, a just launched majority-owned subsidiary of Jaguar Gene Therapy that is focused on creating transformative therapies for ciliopathies, will develop the program.

“We have established an initial preclinical pipeline of AAV9-based gene therapy programs targeting diseases with significant unmet need,” said Joe Nolan, CEO of Jaguar Gene Therapy, who says his team’s experience with the proven and reliable AAV9 platform will minimize development risk and enable the company to accelerate the availability of life-changing therapies to patients, caregivers and their families.

In addition to the Series A funding, Deerfield Management is providing Jaguar Gene Therapy with access to its existing network of 18 key academic institutions. This network allows for the creation of relationships with top academic experts and the ability for Jaguar Gene Therapy to access a variety of potential future gene therapy pipeline opportunities from these world-class laboratories.

Photo: Joe Nolan, CEO of Jaguar Gene Therapy

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