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
October 10 2023 - 8:00AM
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-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), today announced that it will present
data on its TSHA-102 program in evaluation for Rett syndrome during
two poster presentations at the European Society of Gene & Cell
Therapy (ESGCT) 30th Annual Congress, taking place in Brussels,
Belgium from October 24-27, 2023.
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: Emdadul
Haque, Ph.D., Director, Translational Sciences, and Fred Porter,
Ph.D., Chief of Staff and Technical Operations Officer, Taysha Gene
TherapiesPoster Session Date/Time: Wednesday,
October 25 at 17:00-18:15 CET and Thursday, October 26 at
20:30-21:30 CETPoster Session: CNS & Sensory
DiseasesPoster Number: P435
Abstract Title: Early safety and efficacy
observations following the first use of TSHA-102 gene therapy in a
patient with Rett SyndromePresenter: Benit Maru,
MBChB, Ph.D., Chief Medical Officer and Head of Clinical
Development, Taysha Gene TherapiesPoster Session
Date/Time: Wednesday, October 25 at 18:15-19:30 CET and
Thursday, October 26 at 19:30-20:30 CETPoster
Session: Accessibility of Gene TherapyPoster
Number: P302
Additional details on the meeting can be found at the ESGCT 30th
Annual Congress website.
About TSHA-102TSHA-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 Food and Drug Administration (FDA) and has
been granted Orphan Drug designation from the European
Commission.
About Rett SyndromeRett 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, European Union and the United
Kingdom.
About Taysha Gene TherapiesTaysha Gene
Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS
disease. With a singular focus on developing curative medicines, we
aim to rapidly translate our treatments from bench to bedside. We
have combined our team’s proven experience in gene therapy drug
development and commercialization with the world-class University
of Texas Southwestern Gene Therapy Program. Together, we leverage
our fully integrated platform with a goal of dramatically improving
patients’ lives. More information is available
at www.tayshagtx.com.
Company Contact:Hayleigh Collins Director, Head
of Corporate CommunicationsTaysha Gene Therapies,
Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communicationscarolyn.hawley@canalecomm.com
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