Taysha Receives FDA Breakthrough Therapy Designation for TSHA-102
Taysha Gene Therapies has provided an update on its Rett gene therapy program via a press release and community letter. See links below.
Highlights include:
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FDA has granted TSHA-102 Breakthrough Therapy Designation, which expedites the development and review of treatments and dugs, based on preliminary safety and efficacy results from the first 12 participants in the REVEAL Phase 1/2 trial.
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Taysha has reached agreement with the FDA on the design of the REVEAL Pivotal Study, which will begin enrolling participants in late 2025. This study will include a 6-month interim analysis to evaluate whether participants gain or regain key developmental milestones.
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The pivotal trial will enroll 15 girls and young women between the ages of 6 to 22 with typical Rett syndrome. Enrollment is expected to begin this quarter.
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A separate study for younger girls (ages 2–6) is also planned.
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Taysha is exploring trial options outside the U.S. and Canada.
RSRT will continue to share updates as this program progresses.
Questions can be directed to patientaffairs@tayshagtx.com
More information is available by reading Taysha’s community letter and press release below.
