Taysha IPO Presentation Deck
SURF1-related Leigh syndrome is an early
onset neurodegenerative disorder
-
Deficiency in the SURF1 gene results in mitochondrial
dysfunction and dysregulation of oxidative
phosphorylation
SURF1 mutations are implicated in Leigh syndrome as
well as Charcot-Marie Tooth 4K
Symptoms appear in late infancy and include episodic
neurological regression, movement disorder, and
respiratory failure
- The estimated prevalence of SURF1 is 300 patients in
the US and EU
AAV9+
IXT
hSURF1+
Intrathecal
delivery
ISIOON TAITOIN
SURFI
PET100
SUCLA
MTFMT
Age of onset t
Age of death
Age at last report
Age of onset C
Age of death
last report
Age of onset HD
Age of death
Age at last report
Age of onset
Age of death
Age at last report
Age of onset
Age of death
Age at last report
Age of onset
Age of death
Age at last report
*
102 244 215 22 241 212 264 274 24 300 42 104
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