Taysha Gene Therapies Announces Two Poster Presentations on TSHA-102 in Rett Syndrome at Upcoming European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress
Taysha Gene Therapies, a leading clinical-stage gene therapy company, is set to present groundbreaking data on its TSHA-102 program for the treatment of Rett syndrome at the prestigious European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress. The event will take place in Brussels, Belgium from October 24-27, 2023.
Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the MECP2 gene, which is vital for brain function. It affects primarily females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies for Rett syndrome.
Taysha Gene Therapies’ TSHA-102 program offers hope for patients with Rett syndrome. TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology. This innovative approach allows for the precise modulation of MECP2 levels in the central nervous system (CNS) on a cell-by-cell basis, without the risk of overexpression.
During the ESGCT Congress, Taysha Gene Therapies will present new preclinical in vitro data supporting the miRARE technology, as well as initial clinical data from the first adult patient treated with TSHA-102 in the REVEAL Phase 1/2 adult trial. These presentations will shed light on the potential of TSHA-102 to address the underlying cause of Rett syndrome and provide early insights into its safety and efficacy.
The company’s commitment to advancing gene therapies for monogenic diseases of the CNS is evident in its collaboration with the University of Texas Southwestern Gene Therapy Program. By combining their expertise in gene therapy drug development and commercialization, Taysha Gene Therapies aims to rapidly translate their treatments from the laboratory to patients in need.
TSHA-102 has already received Fast Track designation, as well as Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). Additionally, it has been granted Orphan Drug designation from the European Commission, highlighting the potential impact of this therapy on the lives of patients with Rett syndrome.
The upcoming presentations at the ESGCT Congress will provide a platform for Taysha Gene Therapies to share their groundbreaking research with the scientific community. Dr. Emdadul Haque and Dr. Fred Porter will present the in vitro data supporting the miRARE technology, while Dr. Benit Maru will discuss the early safety and efficacy observations from the first patient treated with TSHA-102.
Taysha Gene Therapies’ participation in this prestigious event underscores their dedication to improving the lives of patients with monogenic CNS diseases. By harnessing the power of gene therapy and innovative technologies like miRARE, the company is at the forefront of developing potentially transformative treatments for Rett syndrome and other genetic disorders.
As the ESGCT Congress approaches, the scientific community eagerly anticipates the data that Taysha Gene Therapies will present. With their pioneering approach and commitment to eradicating monogenic CNS diseases, the company is poised to make a significant impact in the field of gene therapy.