Taysha IPO Presentation Deck
Fragile X Syndrome is one of the most common
forms of inherited intellectual disability
Mutations in FMR1 result in decreased FMRP, a protein
necessary for normal brain development
Fragile X Syndrome is characterized by a spectrum of
physical, cognitive, and behavioral features including:
- Low muscle tone
Attention deficits and hyperactivity (ADHD)
Behavior issues, shyness, anxiety, poor eye contact
Speech and language delays
The estimated prevalence of Fragile X is 96,000 patients
in the US and EU
AAV9+
IXT
hFMR1
Intrathecal
delivery
mRNA
FMRP
Clinical
Typical
(CGG) <45
Normal
Premutation
(CGG) 55-200
Primary ovarian insufficiency (POI),
fragile X-associated tremor ataxia
syndrome (FXTAS) due to excess
mRNA
DO
Full mutation
(CGG) > 200
X
Fragile X syndrome
due to lack of FMRP
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