Business
Taysha Gene Therapies Highlights Strategic Priorities and Provides 2021 Business Outlook
Expects Phase 1/2 biomarker and preliminary clinical data for TSHA-101 in GM2 gangliosidosis in second half of 2021 and by year-end 2021, respectively Plans
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nExpects Phase 1/2 biomarker and preliminary clinical data for TSHA-101 in GM2 gangliosidosis in second half of 2021 and by year-end 2021, respectively\n\nPlans to initiate a U.S. Phase 1/2 trial for TSHA-101 in GM2 gangliosidosis in second half of 2021 as well as Phase 1/2 trials for TSHA-118 in CLN1, TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome by year-end 2021\n\nAnticipates advancement of four programs into IND/CTA-enabling studies: SLC13A5 haploinsufficiency, Adult Polyglycosan Body Deficiency (APBD), Lafora disease and GM2 AB variant\n\nExpects IND/CTA submission from one of the following programs: SLC13A5 haploinsufficiency, APBD, Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency\n\nAnticipates advancement of four new undisclosed programs into preclinical development focused on neurodevelopmental disorders, genetic epilepsies and neurodegenerative diseases\n\nIntends to advance the development of next-generation technologies including miRARE platform, redosing strategy, mini-gene payloads and novel capsids, to optimize key components of the company’s AAV-based gene therapies\n\nContinues to make progress on internal 187,000 square-foot, 2,000-liter capacity, multi-product cGMP facility located in Durham, NC\n\n DALLAS--(BUSINESS WIRE)--\nTaysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha”), a patient-centric, clinical-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 highlighted its strategic priorities and provided a business outlook for 2021.\n\n“We enter 2021 having built a strong foundation on which to execute our corporate and pipeline objectives. Notably, we expanded our seasoned leadership team and esteemed board of directors steeped in gene therapy development and commercialization expertise, successfully raised funds in our initial public offering, transitioned from a preclinical- to a clinical-stage company, and achieved important progress on R&D initiatives and our three-pillar manufacturing strategy,” said RA Session II, President, Founder and CEO of Taysha. “2021 will be a transformational year as we intend to rapidly advance multiple drug candidates to clinical proof-of-concept, further expand our pl...