Taysha Gene Therapies' Drug in the FDA Approval Process
This section highlights FDA-related milestones and regulatory updates for drugs developed by Taysha Gene Therapies (TSHA).
Over the past two years, Taysha Gene Therapies has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
TSHA-102. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
TSHA-102 FDA Regulatory Timeline and Events
TSHA-102 is a drug developed by Taysha Gene Therapies for the following indication: Rett Syndrome.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- TSHA-102
- Announced Date:
- June 3, 2025
- Indication:
- Rett Syndrome
Announcement
Taysha Gene Therapies, Inc. announced details for three oral presentations reviewing recent updates supporting its TSHA-102 program in clinical evaluation for Rett syndrome, and a Taysha-hosted symposium on the Company's analysis of the natural history study data at the 2025 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting, taking place in Boston from June 9-11, 2025.
AI Summary
Taysha Gene Therapies, Inc. announced that it will deliver three oral presentations at the 2025 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting in Boston, scheduled for June 9–11, 2025. These presentations will review recent updates supporting the TSHA-102 gene therapy program currently under clinical evaluation for Rett syndrome. Expert speakers will present data on safety and efficacy, insights from caregivers regarding developmental milestones, and preclinical findings on vector biodistribution following lumbar intrathecal delivery. Additionally, the company will host a symposium titled "Gain and Regain of Developmental Milestones: How Natural History Insights are Redefining the Therapeutic Development for Rett Syndrome." This event underscores Taysha’s commitment to advancing research, improving patient care, and potentially enhancing treatment pathways for a condition with significant unmet medical needs.
Read Announcement- Drug:
- TSHA-102
- Announced Date:
- May 28, 2025
- Indication:
- Rett Syndrome
Announcement
Taysha Gene Therapies, Inc. announced positive clinical data from Part A of the REVEAL Phase 1/2 adolescent/adult and pediatric trials evaluating TSHA-102 in Rett syndrome.
AI Summary
Taysha Gene Therapies, Inc. announced positive clinical data from Part A of the REVEAL Phase 1/2 trials evaluating TSHA-102 for Rett syndrome. In this study, 100% of the 10 patients aged 6 to 21 years gained or regained at least one key developmental milestone, a notable achievement given that natural history data show such milestones are unlikely to be achieved in patients over six years old. The results showed that the high dose of TSHA-102 provided faster and more consistent benefits compared to the low dose, with improvements confirmed across several clinical outcome measures. Additionally, the treatment was generally well tolerated, with no treatment-related serious adverse events or dose-limiting toxicities observed. These encouraging findings support further development of TSHA-102 and its evaluation in a pivotal trial focusing on adolescents and adults who have reached a developmental plateau.
Read Announcement- Drug:
- TSHA-102
- Announced Date:
- May 28, 2025
- Indication:
- Rett Syndrome
Announcement
Taysha Gene Therapies, Inc. announced details of its planned pivotal Part B trial design for TSHA-102 following written alignment from the U.S. Food and Drug Administration (FDA).
AI Summary
Taysha Gene Therapies, Inc. has announced its planned pivotal Part B trial for TSHA-102 after receiving written alignment from the U.S. Food and Drug Administration. The trial will be a single-arm, open-label study designed for females with Rett syndrome who are in the developmental plateau stage (age ≥6 years). In this study, each patient will act as her own control, and the primary goal is to measure the gain or regain of developmental milestones that are critical for daily functioning. The Company’s analysis of natural history data showed that patients six years and older rarely achieve new developmental gains without treatment, making this trial endpoint both objective and significant. Taysha plans to submit the trial protocol and statistical analysis plan as an amendment to its IND application later this quarter, with trial initiation activities expected to begin in the third quarter of 2025.
Read Announcement- Drug:
- TSHA-102
- Announced Date:
- September 26, 2024
- Indication:
- Rett Syndrome
Announcement
Taysha Gene Therapies, Inc. announced that it will present previously disclosed clinical data on its TSHA-102 program in evaluation for Rett syndrome during an oral presentation at the upcoming 9th World Rett Syndrome Congress, taking place in Queensland, Australia from October 2-5, 2024.
AI Summary
Taysha Gene Therapies, Inc. announced that it will deliver an oral presentation on its TSHA-102 gene therapy program for Rett syndrome at the 9th World Rett Syndrome Congress in Queensland, Australia, scheduled for October 2–5, 2024. The presentation will include previously disclosed clinical data from the REVEAL Phase 1/2 trials—reporting outcomes up to 52 weeks from the adolescent/adult study and initial findings up to 22 weeks from the pediatric study.
Led by Dr. Sukumar Nagendran, the session will highlight data from different dosing cohorts and provide insights into TSHA-102’s potential as a once‑in‑a‑lifetime treatment. Designed to introduce a functional MECP2 gene directly into the central nervous system, TSHA-102 aims to address the underlying genetic cause of Rett syndrome and offer new hope for patients with this challenging neurodevelopmental disorder.
Read Announcement- Drug:
- TSHA-102
- Announced Date:
- June 18, 2024
- Estimated Event Date Range:
- July 1, 2024 - September 30, 2024
- Target Action Date:
- Q3 2024
- Indication:
- Rett Syndrome
Announcement
Taysha Gene Therapies, Inc. announced that IDMC approved Company's request for early advancement to cohort two (high dose) in the REVEAL pediatric trial; dosing expected in Q3 2024 following IDMC review of initial safety data from the first high dose patient in the adolescent and adult trial
AI Summary
Taysha Gene Therapies, Inc. announced a key milestone in its REVEAL pediatric trial for Rett syndrome. The company’s Independent Data Monitoring Committee (IDMC) approved the early advancement to cohort two, which involves administering a higher dose. This decision followed an initial safety review from the first high dose patient in the adolescent and adult trial, where the safety data was encouraging. As a result, dosing for the high dose pediatric cohort is expected to begin in the third quarter of 2024. This early advancement reflects Taysha’s commitment to rapidly advancing its gene therapy program and supports ongoing efforts to evaluate transformative treatment options for patients with Rett syndrome. Upcoming safety and efficacy data from this cohort will provide further insights into the potential benefits of the therapy.
Read Announcement - Drug:
- TSHA-102
- Announced Date:
- June 18, 2024
- Indication:
- Rett Syndrome
Announcement
Taysha Gene Therapies, Inc. announced positive longer-term clinical data from the ongoing REVEAL Phase 1/2 adolescent and adult trial and initial clinical data from the REVEAL Phase 1/2 pediatric trial evaluating TSHA-102 in Rett syndrome.
AI Summary
Taysha Gene Therapies, Inc. has announced promising longer-term clinical data from its ongoing REVEAL Phase 1/2 trial in adolescent and adult patients, along with early clinical data from a pediatric trial evaluating TSHA-102 for Rett syndrome. In the adult trial, patients showed sustained improvements across several clinical areas, such as enhanced motor skills, better communication, normalized sleep, and a reduction in seizures even after tapering steroid treatments. Notably, one adult patient was able to sit unassisted for the first time in over a decade and experienced improved limb movement. Meanwhile, initial pediatric data revealed improvements in hand function, balance, communication, and new developmental gains, including using eye-gaze communication devices and increased mobility. These positive results support the potential of TSHA-102 as a transformative, one-time intrathecal gene therapy for treating the genetic root cause of Rett syndrome.
Read Announcement- Drug:
- TSHA-102
- Announced Date:
- May 2, 2024
- Indication:
- Rett Syndrome
Announcement
Taysha Gene Therapies, Inc announced the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome.
AI Summary
Taysha Gene Therapies, Inc. announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to TSHA-102, a self-complementary, intrathecally delivered AAV9 gene therapy currently in clinical evaluation for Rett syndrome. This designation follows the FDA’s review of safety and efficacy data from the first three patients treated with a low dose of TSHA-102 across their REVEAL Phase 1/2 trials. RMAT designation is an important step as it allows for increased dialogue with senior FDA managers and may expedite the development and review process of TSHA-102. The therapy aims to address the underlying genetic cause of Rett syndrome by delivering a functional copy of the MECP2 gene to cells in the central nervous system, potentially offering a new treatment option for patients and families facing this severe neurodevelopmental disorder.
Read Announcement
Taysha Gene Therapies FDA Events - Frequently Asked Questions
As of now, Taysha Gene Therapies (TSHA) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Taysha Gene Therapies (TSHA) has reported FDA regulatory activity for TSHA-102.
The most recent FDA-related event for Taysha Gene Therapies occurred on June 3, 2025, involving TSHA-102. The update was categorized as "Oral presentation," with the company reporting: "Taysha Gene Therapies, Inc. announced details for three oral presentations reviewing recent updates supporting its TSHA-102 program in clinical evaluation for Rett syndrome, and a Taysha-hosted symposium on the Company's analysis of the natural history study data at the 2025 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting, taking place in Boston from June 9-11, 2025."
Currently, Taysha Gene Therapies has one therapy (TSHA-102) targeting the following condition: Rett Syndrome.
More FDA Event Resources from MarketBeat
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
This page (NASDAQ:TSHA) was last updated on 7/31/2025 by MarketBeat.com Staff
