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Taysha Gene Therapies Receives Orphan Drug Designation for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency From the European Commission
Designation supports unmet need for treatment options for patients with rare form of genetic epilepsy TSHA-105 is first program in Taysha’s pipeline to
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nDesignation supports unmet need for treatment options for patients with rare form of genetic epilepsy\n\nTSHA-105 is first program in Taysha’s pipeline to receive designation from European Commission\n\nNo approved treatments for the underlying cause of 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-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.\n\n“SLC13A5 deficiency leads to a debilitating form of genetic epilepsy in children that results in persistent seizures and developmental delays, requiring constant supervision and care. With no available disease modifying treatments for this disease, we are pleased that TSHA-105 has been granted orphan drug designation from both the FDA and European Commission, highlighting the global need and important potential of TSHA-105 in helping treat this form of epilepsy,” said RA Session II, President, Founder and CEO of Taysha. “We look forward to working with regulatory agencies to advance this promising gene replacement strategy as expeditiously as possible.”\n\nSLC13A5 deficiency is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. As an autosomal recessive disorder, two copies of the mutated gene must be inherited for an infant to be affected. This type of epilepsy manifests as developmental delay, and seizures beginning within the first few days of life. SLC13A5 deficiency is a rare disorder, with an estimated prevalence of 1,900 patients in the United States and in Europe. Current standards of care are anti-seizure medications which only target the symptoms and do not address the underlying cause of the disease.\n\nThe European Commission grants orphan drug designation for medicines being developed for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect fewer than 5 in 10,000 people in the European Union. Orphan designation in the European Union includes benefits such as protocol assistance, reduced re...