Taysha IPO Presentation Deck
CLN1 is a severe neurodegenerative
lysosomal storage disease
Severe, progressive, neurodegenerative lysosomal
storage disease, with no approved treatment
Caused by mutations in the CLN1 gene, encoding
the soluble lysosomal enzyme palmitoyl-protein
thioesterase-1 (PPT1)
- The absence of PPT1 leads to the accumulation of
palmitoylated substrate within the lysosome
Disease onset is typically within 6-24 months, with
progression visual failure, cognitive decline, loss of
fine and gross motor skills, seizures, and death
usually occurring by 7 years of age
The estimated prevalence of CLN1 disease is 600
patients (US+EU)
- Currently an open IND for this program
Enables rapid onset
and stable expression
SCAAV ITR
F.
CBh
Drive high levels of
gene expression
Full copy of hCLN1 gene
hCLNI
*
AAV9 capsid
Brain tropism &
favorable safety profile
SCAAV ITR
Enables rapid onset
and stable expression
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