Open-label, Randomized, Dose-escalation and Dose-expansion Study of the Safety and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, in the Treatment of Adult Females With Rett Syndrome
Latest Information Update: 21 Jun 2024
At a glance
- Drugs TSHA 102 (Primary)
- Indications Rett syndrome
- Focus Adverse reactions; First in man; Therapeutic Use
- Acronyms REVEAL; REVEAL Adult Study
- Sponsors Taysha Gene Therapies
- 18 Jun 2024 According to a Taysha Gene Therapies media release, initial available safety and efficacy data from cohort two expected in the second half of 2024.
- 18 Jun 2024 According to a Taysha Gene Therapies media release, first patient has been dosed in cohort two (high dose, n=3) of 1x1015 total vg in the second quarter of 2024
- 18 Jun 2024 Results presented in a Taysha Gene Therapies Media Release.