Taysha Gene Therapies R&D Day 1


06/28/2021
10:00 am - 1:00 pm EDT

Topics of discussion will include:

-TSHA-120 (GAN): Pivotal-stage AAV9 gene replacement therapy program for the treatment of giant axonal neuropathy (GAN), a rare autosomal recessive disease of the central and peripheral nervous systems caused by loss-of-function gigaxonin gene mutations.
-TSHA-101 (GM2 gangliosidosis): The first bicistronic AAV9 gene therapy in clinical development designed to deliver two genes, HEXA and HEXB, comprising the alpha and beta sub-units of beta hexosaminidase A, intrathecally for the treatment of GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease. TSHA-101 is currently in Phase 1/2 development.
-TSHA-118 (CLN1 disease): AAV9-based gene therapy designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1 disease, a rapidly progressing rare lysosomal storage disease with no approved treatments. This gene replacement therapy program is currently under an open IND, with initiation of a Phase 1/2 trial expected in the second half of 2021.
-TSHA-102 (Rett syndrome): AAV9-based gene therapy in development for Rett syndrome, a severe neurodevelopmental disorder, designed to deliver MECP2, as well as a novel miRARE platform that regulates transgene expression on a cell-by-cell basis. This regulated gene replacement therapy is currently in IND/CTA-enabling studies, with an IND/CTA filing expected in the second half of 2021.