Taysha Gene Therapies to Present Biodistribution Data from an Analysis Evaluating AAV9 Gene Therapy Delivery at the Upcoming 31st Annual ESGCT Congress
TSHA 10.22.2024
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Data from an analysis of five NHP studies showed lumbar IT administration led to widespread and consistent biodistribution of AAV9 gene therapy vectors across brain and spinal cord regions
IT and ICM administration achieved comparable biodistribution across brain and spinal cord regions in NHPs
Findings from the analysis reaffirm the clinical potential of IT administration as an effective, safe and minimally invasive approach to deliver AAV-based gene therapies designed to treat CNS diseases in both children and adults
“Findings from an analysis across five NHP studies showed that both IT and ICM administration led to comparable, consistent and widespread biodistribution of AAV9 vector throughout the brain and spinal cord regions,” said
Poster presentation details are as follows:
Title: Broad CNS Biodistribution of AAV9-based Gene Therapies Delivered by Intrathecal Lumbar Puncture in Non-Human Primates
Presenters:
Date and Time:
Poster Session: CNS & Sensory Diseases
Poster Number: P0284
Additional details on the meeting can be found at the 31stAnnual
About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form ofMECP2to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels ofMECP2in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the
About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linkedMECP2gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. 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 pathogenicMECP2mutation is estimated to affect between 15,000 and 20,000 patients in the
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Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include, but are not limited to, statements concerning the potential of TSHA-102 and Taysha’s other product candidates to positively impact quality of life and alter the course of disease in the patients Taysha seeks to treat, its research, development and regulatory plans for its product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed and the potential market opportunity for Taysha’s product candidates. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding Taysha’s business are described in detail in its SEC filings, including in Taysha’s Annual Report on Form 10-K for the full-year ended December 31, 2023 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, which are available on the SEC’s website atwww.sec.gov. Additional information will be made available in other filings that Taysha makes from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and Taysha disclaims any obligation to update these statements except as may be required by law.
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Source: Taysha Gene Therapies, Inc.