Jaguar Gene Therapy Announces Preclinical Data Presentations at Upcoming American Society of Gene and Cell Therapy 25th Annual Meeting

LAKE FOREST, Ill.--(BUSINESS WIRE)-- Jaguar Gene Therapy, a biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including those that affect sizeable patient populations, today announced two abstracts related to its JAG101 Type 1 galactosemia program will be presented at the American Society of Gene and Cell Therapy (ASGCT) 25^th Annual Meeting, which is taking place at the Walter E. Washington Convention Center in Washington, D.C., from May 16-19. JAG101, an investigational gene therapy currently in preclinical development, is intended as a one-time treatment for Type 1 galactosemia.

“Jaguar successfully executed two different animal studies at two different institutions, and the data from both support the continued development of JAG101 as a one-time treatment for Type 1 galactosemia, a rare genetic disease for which no treatments are FDA-approved,” said Suku Nagendran, M.D., President of R&D and Chief Medical Officer at Jaguar Gene Therapy. “These promising animal studies conducted in partnership with researchers at Emory University and the University of Utah give us confidence in our approach to develop JAG101 as a gene replacement solution designed to address the root cause of Type 1 galactosemia by delivering the functional GALT gene via the AAV9 vector. We believe gene therapy could potentially prevent the buildup of galactose, Gal-1P and galactitol and their potential contribution to long-term complications, to positively change the clinical course of this disease.”

Both abstracts are now available at https://annualmeeting.asgct.org/abstracts. Additional data beyond that included in the abstracts will be presented at the meeting. Details of the data presentations follow:

Wednesday, May 18, 5:30-6:30 PM ET
Poster Session: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases II
Poster Board #: W-132
Abstract title: Gene replacement therapy with JAG101 reduces pathogenic biomarkers in a mouse model of Type 1 galactosemia
Abstract #: 1006
Presenter: Sandeep Dhall, Ph.D., Associate Director, Preclinical Programs, Jaguar Gene Therapy

Wednesday, May 18, 5:30-6:30 PM ET
Poster Session: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases II
Poster Board #: W-134
Abstract title: Gene replacement with JAG101 leads to GALT transgene expression in target organs and reduces toxic metabolites in a rat model of Type 1 galactosemia
Abstract #: 1008
Presenter: Michael Hughes, Ph.D., Associate Director, Research, Jaguar Gene Therapy

About Type 1 Galactosemia
Type 1 galactosemia is a rare genetic disease that can be life-threatening for newborns and cause severe lifelong complications starting as early as the first year of life.^1,2,3 Galactosemia affects the body’s ability to make the enzyme that breaks down galactose, a simple sugar the body endogenously produces and is also found in dairy and other foods, including breast milk.^1,4,5 Type 1 galactosemia is caused by mutations in the GALT gene, which lead to a severe deficiency in functional galactose-1-phosphate uridylyltransferase (GALT) enzyme, which causes a toxic buildup of galactose and its metabolites including Gal-1P and galactitol. This buildup of toxic metabolites is a life-threatening medical emergency in newborns and can contribute to lifelong cognitive, neurological, and speech complications, as well as primary ovarian insufficiency in girls and women.^1,2,4,5 Because of its severity, galactosemia is part of newborn screening in all 50 states of the United States and in several other countries.^1,2,5 No treatments are currently approved for galactosemia, and there is significant unmet medical need. The current standard of care – a galactose-restricted diet – is insufficient because the body endogenously produces galactose, causing patients to experience chronic complications.²

To learn more about Type 1 galactosemia, view the Roundtable Discussion Understanding Type 1 Galactosemia.

About JAG101
JAG101 aims to deliver a gene replacement solution to address the root cause of Type 1 galactosemia by delivering the functional GALT gene via the AAV9 vector. Gene therapy offers the opportunity to have immediate impact to reduce multiple toxic metabolites simultaneously, notably galactose, Gal-1P and galactitol, bringing them closer to a normal level to positively change the clinical course of the disease and mitigate longer-term complications. Jaguar has research agreements in place for the program with Emory University and the University of Utah, which produced encouraging preclinical proof-of-concept data in animals.

About Jaguar Gene Therapy
Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable patient populations. The company is made up of a proven team of experts with unparalleled CMC (Chemistry, Manufacturing and Controls), regulatory, clinical and commercial acumen who have first-hand experience in bringing novel gene therapy treatments to patients and their families. Committed to patient safety and product purity, Jaguar is rapidly advancing an initial pipeline of three programs targeting: 1) Type 1 galactosemia; 2) a genetic cause of autism spectrum disorder and Phelan-McDermid syndrome and other severe neurodevelopmental disorders with a SHANK3 mutation or deletion; and 3) Type 1 diabetes. The company continues to evaluate opportunities to expand its pipeline using the strength of the team and close relationships with numerous academic institutions. For more information, please visit www.jaguargenetherapy.com and follow Jaguar Gene Therapy on LinkedIn.

References
¹Galactosemia. National Organization for Rare Disorders (NORD) Rare Disease Database. 2019. Accessed October 27, 2021. https://rarediseases.org/rare-diseases/galactosemia/

²Berry GT. Classic galactosemia and clinical variant galactosemia. February 4, 2000. Updated March 11, 2021. Accessed October 27, 2021. https://www.ncbi.nlm.nih.gov/books/NBK1518/

³Rubio-Gozalbo ME, Gubbels CS, Bakker JA, Menheere PPCA, Wodzig WKWH, Land JA. Gonadal function in male and female patients with classic galactosemia. Hum Reprod Update. 2010;16(2):177-188. https://doi.org/10.1093/humupd/dmp038

⁴GALT gene. MedlinePlus. August 18, 2020. Accessed October 27, 2021. https://medlineplus.gov/genetics/gene/galt/

⁵Galactosemia. Genetic and Rare Diseases (GARD) Information Center. 2021. Accessed October 27, 2021. https://rarediseases.info.nih.gov/diseases/2424/galactosemia

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