Jaguar Gene Therapy Presents Preclinical Data of JAG101 in Type 1 Galactosemia at American Society of Gene and Cell Therapy 25th Annual Meeting
AKE FOREST, Ill., May 16, 2022 – 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 results of two preclinical studies of JAG101 in two different animal models of Type 1 galactosemia. Results showed that low and high doses of intravenous JAG101 given as a one-time gene replacement therapy reduced levels of toxic metabolites in brain and liver tissues and increased GALT enzyme expression and activity in tissues relevant to long-term outcomes in Type 1 galactosemia.