Jaguar Gene Therapy Closes $139 Million Series B Funding Co-led by Eli Lilly and Company and Deerfield Management

“We are thrilled to have attracted this prestigious group of visionary investors, which affirms our strategic approach, promising pipeline programs, and unique collaboration with Deerfield Management,” said Joe Nolan, Chief Executive Officer of Jaguar Gene Therapy. “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.”

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.

“We are excited to engage in Jaguar Gene Therapy’s endeavor to develop novel gene therapies, following the breaking science in this rapidly advancing field,” said Andrew Adams, Ph.D., Vice President of New Therapeutic Modalities at Lilly. “With this investment, we hope Jaguar can accelerate the advancement of a diverse set of innovative medicines for patients.”

Jaguar Gene Therapy will use the proceeds of the Series B funding, together with its existing cash resources, to advance its initial pre-clinical 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.

“We are proud to invest in Jaguar Gene Therapy from our Fund XI, which was launched this year to support early-stage biotechnology companies that aim to meaningfully change medicine for the better,” said Robert Nelsen, Managing Director at ARCH Venture Partners. “Having worked closely with the Jaguar senior team members when they were leading AveXis, I have first-hand experience of their expertise, creativity, flexibility, resilience and proven track record of success in gene therapy development, manufacturing and commercialization.”

About Jaguar Gene Therapy

Jaguar Gene Therapy, LLC is accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases. 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 more information, please visit www.jaguargenetherapy.com and follow Jaguar Gene Therapy on LinkedIn.

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