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
- Taysha
Taysha Gene Therapies Provides Update on TSHA-120 Program in Giant Axonal Neuropathy and a 2023 Corporate Outlook
- Neurogene
Neurogene Announces FDA Clearance of IND for NGN-401 Gene Therapy for Children with Rett Syndrome
- Taysha
Astellas and Taysha Gene Therapies Announce Strategic Investment to Support Development of Taysha’s AAV-based Gene Therapy Programs
- Acadia
Acadia Pharmaceuticals Announces Trofinetide New Drug Application for the Treatment of Rett Syndrome has been Accepted for Filing and Review by U.S. FDA
- Acadia
Acadia Pharmaceuticals Submits New Drug Application to the U.S. FDA for Trofinetide for the Treatment of Rett Syndrome
- Alcyone
Alcyone Presents Preclinical Data on Gene Therapy Programs with The Center for Gene Therapy at The Abigail Wexner Research Institute (AWRI) at ASGCT 2022
- Taysha
Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-102 in Rett Syndrome
- Stoke Acadia
Acadia Pharmaceuticals and Stoke Therapeutics Announce Collaboration to Pursue Multiple RNA-based Treatments for Severe and Rare Genetic Neurodevelopmental Diseases
- Acadia
Acadia Pharmaceuticals Announces Positive Top-line Results from the Pivotal Phase 3 Lavender Trial of Trofinetide in Rett Syndrome
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