Taysha IPO Presentation Deck slide image

Taysha IPO Presentation Deck

Deep pipeline of gene therapies targeting neurodevelopmental disorders TSHA-106 shRNA Angelman syndrome Preclinical TSHA-114 GRT Fragile X syndrome Preclinical TSHA-116 shRNA Prader-Willi syndrome Preclinical TSHA-117 regulated GRT FOXG1 syndrome Preclinical • Imprinting disorder in which the maternal gene is deficient and the paternal copy of UBE3A is intact but silenced by a long non-coding RNA • • . . . The disease causes profound developmental delay, ataxia and gait disturbance Estimated prevalence of 30,000 patients in the US and EU FMR1 is the most common single gene cause of autism and cognitive impairment Fragile X Syndrome is characterized by anxiety, aggression, hyperactivity, attention deficits, and sleep/communication disruption Estimated prevalence of 96,000 patients in the US and EU Loss of function of genes along 15q11-q13 chromosome region due to an imprinting defect Patients have developmental delay, insatiable eating habits accompanied by obesity and overt diabetes Estimated prevalence of 40,000 patients in the US and EU Newly discovered gene with prevalence expected to steadily rise as more children as tested with autism spectrum disorder Development and intellectual disabilities, growth restriction with microcephaly, epilepsy, and hyperkinetic-dyskinetic movement disorder Estimated prevalence of 20,000 patients in the US and EU T 00 38
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