TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. The Company will present new preclinical in vitro data supporting the miRARE technology, as well as initial clinical data from the first adult patient dosed with TSHA-102 in the REVEAL Phase 1/2 adult trial.
Poster presentation details are as follows:
Abstract Title: The microRNA-responsive autoregulatory element from TSHA-102 for Rett Syndrome modulates therapeutic transgene expression in response to cellular MECP2 in mouse and human cell lines
Presenters:
Poster Session Date/Time:
Poster Session: CNS & Sensory Diseases
Poster Number: P435
Abstract Title: Early safety and efficacy observations following the first use of TSHA-102 gene therapy in a patient with Rett Syndrome
Presenter:
Poster Session Date/Time:
Poster Session: Accessibility of Gene Therapy
Poster Number: P302
Additional details on the meeting can be found at the ESGCT 30th Annual
About TSHA-102
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Fast Track designation and Orphan Drug and Rare Pediatric Disease designations from the
About Rett Syndrome
Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene, which is a gene that’s essential for neuronal and synaptic function in the brain. The disorder is characterized by intellectual disabilities, loss of communication, seizures, slowing and/or regression of development, motor and respiratory impairment, and shortened life expectancy. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the Unites States,
About
Company Contact:
Director, Head of Corporate Communications
hcollins@tayshagtx.com
Media Contact:
carolyn.hawley@canalecomm.com
Source: Taysha Gene Therapies, Inc.