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
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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
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