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Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis
TSHA-101 is the first and only bicistronic vector being developed in a Phase 1/2 clinical study to date Preliminary clinical safety and biomarker data from
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nTSHA-101 is the first and only bicistronic vector being developed in a Phase 1/2 clinical study to date\n\nPreliminary clinical safety and biomarker data from the Queen’s University study expected by year-end\n\nNo approved treatments for the disease\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 has been granted orphan drug designation from the European Commission for TSHA-101, an AAV9-based bicistronic gene replacement therapy in development for GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease.\n\n“GM2 gangliosidosis is a fatal neurodegenerative disease caused by deficiency in the lysosomal enzyme β-hexosaminidase A, also known as Hex A. The prognosis is devastating, with infantile forms often leading to death within the first four years of life and juvenile onset patients rarely surviving beyond mid-teens,” said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. “Residual Hex A activity correlates with the severity of GM2, and based on our understanding of this correlation, small increases in Hex A activity are likely to lead to significant improvements in clinical outcomes and quality of life. Based on dose-dependent improvements in survival in preclinical models, we are highly encouraged that our novel bicistronic gene therapy approach with TSHA-101 has the potential to be a life changing therapy for patients suffering from this rapidly progressive disorder with no current treatment options.”\n\nGM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder that is part of 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 (Hex A) enzyme. These genetic defects result in progressive dysfunction of the central nervous system. Residual Hex A enzyme activity determines the severity of the disease. The infantile form of the disease has an onset of symptoms usually before six months of age with ...