Clinical EffectivenessTSHA's gene therapy program has shown promising results with continued patient benefits and no plateau in improvements, especially in high-dose treatments.
Commercial OpportunityDr. Lieberman appeared to find clinical data to date for TSHA-102 promising, and there is more positivity on the commercial opportunity for gene therapy in Rett syndrome.
Regulatory FlexibilityAnnounced FDA alignment for pivotal TSHA-102 trial shows continued flexibility.