News from the Rett Syndrome Pipeline
Press releases, community letters, and more from the companies advancing towards treatments and cures for Rett syndrome.
June 19, 2025
Taysha Gene Therapies Updates on Part A Data, Plans for the Part B Pivotal Study, and FAQs (Community Letter)
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- Neurogene
Neurogene Updates on Program & Risk Mitigation Strategies (Community Letter)
- Taysha
Taysha Gene Therapies Announces Pivotal Part B Trial Design Details for TSHA-102 in Rett Syndrome Enabled by IRSF Natural History Data and Positive Clinical Data from Part A of the REVEAL Adult/Adolescent and Pediatric Trials Evaluating TSHA-102
- Acadia
Acadia Pharmaceuticals Announces Recipients of Second Annual Rett Sibling Scholarship
- Taysha
Taysha Gene Therapies Reports First Quarter 2025 Financial Results and Provides Corporate Update
- Neurotech
European Commission grants NTI64 Orphan Drug Designation for Rett Syndrome
- Neurogene
Neurogene Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Updates
- Acadia
Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med
- Minoryx
Minoryx announces dosing of the first patient with leriglitazone in the phase 2a study in Rett syndrome (TREE study)
- Taysha
Taysha Gene Therapies Reports Full-Year 2024 Financial Results and Provides Corporate Update
- Unravel
Unravel Announces Collaboration with Vanderbilt University Medical Center to Support Upcoming Multicenter U.S. Clinical Study in Rett Syndrome that Includes RVL-001
- Neurogene
Neurogene Receives Australian HREC Approval for Trial
- Neurogene
Neurogene Presents Favorable Safety Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome during ASGCT Annual Meeting
- Neurotech
Neurotech Reports Significant Clinical Benefits and Safety in Phase I/II Rett Syndrome Clinical Trial
- Taysha
Taysha Gene Therapies Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by U.S. FDA for TSHA-102 in Rett Syndrome
- Acadia
Acadia Pharmaceuticals Announces Trofinetide New Drug Submission for Treatment of Rett Syndrome Has Been Accepted for Filing and Priority Review by Health Canada
- Unravel
Unravel Announces Initiation of RVL001 Clinical Trial Material Manufacturing to Support Upcoming US and International Clinical Trials in Rett Syndrome
- DepYmed
DepYmed Receives Clearance from U.S. Food and Drug Administration to Initiate Phase 1 Clinical Trial for DPM-1003 for the Treatment of Rett Syndrome
- Taysha
Taysha Shares Interim Clinical Data from Cohort One on FY23 Financial Results Call
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