TSHA-101 to be first bicistronic vector evaluated in human clinical trials; TSHA-101 designed to deliver both
TSHA-101 CTA is the second clinical trial clearance received, in addition to TSHA-118’s open investigational new drug application for CLN1
Interim data from Phase 1/2 trial anticipated in 2021
“TSHA-101 will be the first bicistronic vector to enter a first-in-human clinical study, which is a significant milestone for Taysha and for the field of gene therapy,” said
The trial will be a single arm, open-label Phase 1/2 trial evaluating the use of TSHA-101 for the treatment of infants with GM2. The study will be sponsored by Queen’s University and led by
“Preclinical evidence to date supports our belief that TSHA-101, when given intrathecally as a bicistronic transgene packaged into a single AAV9 vector, has the potential to address the lysosomal enzyme deficiency, to change the disease trajectory and to improve patient survival,” said Dr.
“Today’s CTA approval is a culmination of our team’s and Dr. Walia’s tireless efforts and a momentous occasion for children affected by GM2 along with their parents and caregivers,” said RA Session II, Founder, President and CEO of Taysha. “We are grateful to our partners at Queen’s University for their work to advance this gene therapy into the clinic.”
About GM2 Gangliosidosis
GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the β-hexosaminidase A enzyme. These genetic defects result in progressive dysfunction of the central nervous system. There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.
TSHA-101 is an investigational gene therapy administered intrathecally for the treatment of infantile GM2 gangliosidosis. The gene therapy is designed to deliver two genes –
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