Jaguar Gene Therapy Launches with Mission to Accelerate Breakthroughs in Gene Therapy for Patients Suffering from Severe Genetic Diseases

• New venture is led by the former AveXis management team and was created in collaboration with Deerfield Management, which funded the Series A financing and provides access to established academic institutions for future pipeline programs
• The current 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
• Jaguar leverages a proven management team, access to capital and world-class clinical and AAV GMP manufacturing expertise to expedite development timelines

LAKE FOREST, Ill. (Feb. 25, 2021) – Jaguar Gene Therapy launched today after being in stealth mode for the past year and is uniquely positioned to accelerate gene therapy development. Led by the former AveXis leadership and resourced by Deerfield Management, 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.

“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 bench to bedside as safely and quickly as possible.

Jaguar Gene Therapy is focused on advancing 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.
• 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. This program is being developed by Axovia Therapeutics, a majority-owned subsidiary of Jaguar Gene Therapy that is focused on creating transformative therapies for ciliopathies.

“We have established an initial pre-clinical pipeline of AAV9-based gene therapy programs targeting diseases with significant unmet need, including galactosemia, genetic causes of autism spectrum disorder, Type 1 diabetes and Bardet-Biedl syndrome,” said Joe Nolan, Chief Executive Officer of Jaguar Gene Therapy. “I am confident that our team’s experience with the proven and reliable AAV9 platform will minimize development risk and enable us 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.

“The possibility of transforming genetic diseases is now a reality, and Deerfield is excited to be working again with the team we partnered with so successfully at AveXis to develop best-in-class gene therapy treatments,” said James Flynn, Managing Partner at Deerfield Management. “The unique access that we provide to Jaguar via our established Deerfield research collaborations will continue to expand Jaguar’s pipeline. We are proud to be part of this effort and look forward to solving the unmet needs of thousands of patients.”

About Jaguar Gene Therapy

Jaguar Gene Therapy, LLC is accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases. Backed by Deerfield Management, a healthcare-specific investment firm, Jaguar brings streamlined R&D, manufacturing and clinical operations to accelerate development and potential approval of therapies for severe genetic diseases. For more information, please visit http://www.jaguargenetherapy.com/ and follow Jaguar Gene Therapy on LinkedIn.

About Axovia Therapeutics

Axovia is dedicated to developing disease-transformative therapies that target key aspects of ciliopathies, addressing the devastating impact of the disease head-on for patients, their families and caregivers. Axovia is a majority-owned subsidiary of Jaguar Gene Therapy.

About Deerfield Management Company

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. For more information, please visit www.deerfield.com.

Media Contact

Julie Normart
media@jaguargenetherapy.com
559.974.3245

Company Contact

Kate Neer
kneer@jaguargenetherapy.com
815.978.3891