RARE Daily

Jaguar Gene Therapy Closes $139 Million Funding to Advance Preclinical Rare Disease Pipeline

April 13, 2021

Rare Daily Staff

Less than two months after its public launch, Jaguar Gene Therapy said it has closed a $139 million series B funding to accelerate the development of its preclinical pipeline targeting severe genetic diseases.

Eli Lilly and Company and Deerfield Management co-led the financing, with participation from ARCH Venture Partners, Goldman Sachs, and Nolan Capital, the investment fund of former AveXis CEO and current Jaguar Executive Chairman Sean Nolan.

“With this capital infusion, we are well positioned to execute on our mission to accelerate breakthroughs in gene therapy and bring them to children and adults with severe genetic diseases,” said Joe Nolan, CEO of Jaguar Gene Therapy.

Led by former AveXis leadership, Jaguar leverages a proven management team with the expertise to accelerate the development, manufacturing and commercialization of novel gene therapy treatments for patients suffering from severe genetic diseases, including galactosemia, genetic causes of autism spectrum disorder, type 1 diabetes, and Bardet-Biedl syndrome.

Jaguar Gene Therapy will use the proceeds of the series B funding, together with its existing cash resources, to advance its initial preclinical pipeline of AAV9-based gene therapies.

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.

Additionally, Axovia Therapeutics, a majority-owned subsidiary of Jaguar Gene Therapy that is focused on creating transformative therapies for ciliopathies, is advancing AXV101, a gene therapy treatment for BBS1, a subset of Bardet-Biedl syndrome (BBS). A life-threatening neurometabolic condition, BBS causes progressive vision loss, severe obesity, learning disorders and kidney disease.

Photo: Joe Nolan, CEO of Jaguar Gene Therapy

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