Business
Taysha Gene Therapies to Host Virtual R&D Day
Two-day R&D day on June 28 and 29, 2021 at 10:00 am ET will highlight progress across its pipeline and will feature presentations from key opinion leaders
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nTwo-day R&D day on June 28 and 29, 2021 at 10:00 am ET will highlight progress across its pipeline and will feature presentations from key opinion leaders\n\n DALLAS--(BUSINESS WIRE)--\nTaysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will host its first two-day virtual research and development (R&D) day for analysts and investors. The event will be webcast live on June 28 and June 29, 2021 from 10:00 a.m. to 1:00 p.m. ET each day.\n\nThe event will highlight the company’s R&D progress, focused on advancement of its early- and late-stage investigational programs. Topics of discussion will include:\n\nDay 1 – June 28, 2021\n\n\nTSHA-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.\n\n\nTSHA-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.\n\n\nTSHA-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 currently 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.\n\n\nTSHA-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.\n\n\nDay 2 – June 29, 2021\n\n\nTSHA-104 (SURF1-associated Leigh syndrome): AAV9-base...