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Taysha Receives Orphan Drug Designation from the European Commission for TSHA-102 for the Treatment of Rett Syndrome
Preclinical data provide quantitative evidence of TSHA-102’s ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nPreclinical data provide quantitative evidence of TSHA-102’s ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions on a cell-by-cell basis\n\nSignificant survival benefit demonstrated in 4-5 week-old TSHA-102-treated knockout Rett mice with meaningful accumulated disease, a more translatable model of the disorder in humans\n\nIND/CTA filing expected in second half of 2021, with Phase 1/2 trial initiation anticipated by year-end\n\nEstimated 25,000 patients in U.S. and Europe represent multi-billion-dollar commercial opportunity\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-102, an AAV9-based gene replacement therapy in development for Rett syndrome.\n\n“The receipt of orphan drug designation from the European Commission represents an important regulatory milestone that has the potential to expedite the global clinical development of TSHA-102, a one-time gene therapy with disease modifying potential,” said RA Session II, President, Founder and Chief Executive Officer of Taysha. “Promising preclinical data demonstrate that TSHA-102’s novel self-regulatory feedback mechanism, miRARE, has the ability to regulate MECP2 expression in a genotype-dependent manner on a cell-by-cell basis. We are highly encouraged that this novel treatment approach can help effectively address a disease that has historically been difficult to treat, and we look forward to submitting an IND/CTA in the second half of this year and initiating a Phase 1/2 clinical trial by year-end.”\n\nRett syndrome is a severe genetic neurodevelopmental disorder caused by a mutation in the X-linked MECP2 gene essential for neuronal and synaptic function in the brain. Primarily occurring in females, Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 in the United States and European Union. Patients have normal early development, with symptom onset typically beginning between s...