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Introductions
RA Session II
President, Founder & CEO
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TSHA-104
SURF1 deficiency
TSHA-104 for
SURF1-Associated
Leigh Syndrome
Suyash Prasad, MBBS, MSc, MRCP, MRCPCH, FFPM
Chief Medical Officer and Head of R&D
Steven Gray, PhD
Chief Scientific Advisor, UTSW Gene Therapy Program
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SURF1 deficiency is the most common cause of Leigh syndrome
A monogenic mitochondrial disorder
Most common cause of cytochrome c oxidase deficient Leigh syndrome
Leigh syndrome - severe neurological disorder that presents in the first year of life
Initially often presents with gastrointestinal symptoms
Progressive loss of mental and movement abilities, often regression is episodic in nature
Can result in death within two to three years
~10-15%have SURF1 mutation
No approved therapies
Estimated prevalence of SURF1 deficiency is 300 to 400 patients (US+EU)
TSHA-104
SURF1 deficiency
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Taysha Gene Therapies Inc. published this content on 29 June 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 30 June 2021 10:21:03 UTC.
Taysha Gene Therapies Inc is a clinical-stage biotechnology company, which is focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. The Companyâs lead clinical program, TSHA-102, is in development for the treatment of Rett syndrome, a rare neurodevelopmental disorder. The Company is evaluating TSHA-102 in the REVEALPhase I/II adolescent and adult clinical trial, which is a first-in-human, open-label, randomized, dose escalation and dose-expansion, multicenter study evaluating the safety and preliminary efficacy of TSHA-102 in female patients aged 12-years and older with Rett syndrome. It has acquired a worldwide right to a clinical-stage, intrathecally dosed AAV9 gene therapy program, TSHA-120, for the treatment of giant axonal neuropathy (GAN). It received orphan drug designation from the European Commission for TSHA-120 for the treatment of GAN.
TAYSHA GENE THERAPIES, INC. 
