Drugs / LONIMECGENE RENPARVOVEC
LONIMECGENE RENPARVOVEC
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT06152237 | Dec 2023 → Nov 2028 expected | Rett syndrome | Taysha Gene Therapies, Inc. | Active not recruiting | No outcome recorded |
| Phase 32 trials · 1 met primary | ||||||
| Phase 3 | NCT07480564 | May 2026 → Jun 2031 expected | Rett syndrome | Taysha Gene Therapies, Inc. | Recruiting | No outcome recorded |
| Phase 3 | NCT05606614 | Mar 2023 → Jun 2031 expected | Rett syndrome | Taysha Gene Therapies, Inc. | Active not recruiting | Met primary |
News releases announcing trial results or a regulatory action · 22
| Date | Issuer | Release |
|---|---|---|
| 2026-06-25 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Announces Multiple Presentations Highlighting its TSHA-102 Clinical Program at the 2026 IRSF Rett Syndrome Scientific Meeting tayshagtx.com ↗
Recently disclosed longer-term REVEAL Part A data demonstrated broad, multi-domain functional impact that deepened over time through ≥12 months post-TSHA-102 regardless of age or disease severity |
| 2026-06-22 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Announces Completion of Dosing in REVEAL Pivotal Trial and Reports Longer-Term Clinical Data from Part A of REVEAL Phase 1/2 Trials Evaluating TSHA-102 for Rett Syndrome tayshagtx.com ↗
TSHA-102 was generally well-tolerated with no treatment-related SAEs or DLTs reported as of the June 2026 data cutoff across REVEAL Phase 1/2 and pivotal trials (N=29) |
| 2026-04-27 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies to Present New Preclinical Data Supporting Construct Design of TSHA-102 for Rett Syndrome at the ASGCT 2026 Annual Meeting tayshagtx.com ↗
In vitro data demonstrated self-complementary AAV9 (scAAV9) enabled ~30-fold higher MeCP2 protein expression compared to single-stranded AAV9 (ssAAV9), supporting the ability to effectively deliver TSHA-102 to the CNS by lumbar IT administration |
| 2025-10-09 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Presents New Supplemental Data Analysis from Part A of the REVEAL Phase 1/2 Trials for TSHA-102 in Rett Syndrome at the 54th CNS Annual Meeting tayshagtx.com ↗
The analysis provides supportive evidence of additional functional gains in skills and improvements across core disease characteristics that are outside of the natural history defined developmental milestones, further highlighting the consistent, multi-domain impact of TSHA-102 on activities of daily living. |
| 2025-10-02 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Announces FDA Breakthrough Therapy Designation and Provides Positive Regulatory Update on TSHA-102 in Rett Syndrome tayshagtx.com ↗ |
| 2025-05-28 | Taysha Gene Therapies, Inc. | Results 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 tayshagtx.com ↗
Additionally, the Company announced positive clinical data from Part A of the REVEAL Phase 1/2 adolescent/adult and pediatric trials evaluating TSHA-102 in Rett syndrome. |
| 2024-06-18 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Announces Positive Clinical Data Across Adult and Pediatric Patients from Low Dose Cohort in Ongoing REVEAL Phase 1/2 Trials Evaluating TSHA-102 in Rett Syndrome tayshagtx.com ↗ |
| 2024-05-02 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by U.S. FDA for TSHA-102 in Rett Syndrome tayshagtx.com ↗
today 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. |
| 2024-03-19 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Reports Full Year 2023 Financial Results and Provides Corporate and Clinical Updates tayshagtx.com ↗
Data from first adult patient in REVEAL Phase 1/2 trial showed TSHA-102 (low dose, 5.7x1014 total vg) was well-tolerated with no treatment-emergent SAEs as of 35-week assessment |
| 2023-11-29 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Announces Expanded Eligibility in REVEAL Phase 1/2 Adult Trial to Include Adolescent Rett Syndrome Patients tayshagtx.com ↗
Health Canada has authorized the protocol amendment to the ongoing REVEAL Phase 1/2 adult trial evaluating TSHA-102 that expands eligibility to include patients aged 12 and older with Rett syndrome. |
| 2023-11-14 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Reports Third Quarter 2023 Financial Results and Provides Corporate and Clinical Updates tayshagtx.com ↗
Data from second adult patient showed TSHA-102 was well-tolerated with no treatment-emergent SAEs as of six-week assessment with improvement across key efficacy measures, including CGI-I, R-MBA, PGI-I and RSBQ at week four |
| 2023-10-24 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Presents New Preclinical In-vitro Data on TSHA-102 in Rett Syndrome Supporting miRARE Regulation of MECP2 Expression at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress tayshagtx.com ↗
In vitro data demonstrated the miRARE control element downregulates MECP2 transgene and protein expression in response to cellular levels of MeCP2 in cell culture models |
| 2023-08-24 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Announces Fast Track Designation Granted by U.S. FDA for TSHA-102 in Rett Syndrome tayshagtx.com ↗
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced the U.S. FDA has granted Fast Track Designation (FTD) to TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome. |
| 2023-08-14 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Reports Initial Clinical Data from First Adult Rett Syndrome Patient Dosed in REVEAL Phase 1/2 Trial and Provides Corporate Update with Second Quarter 2023 Financial Results tayshagtx.com ↗
Data from first adult patient dosed in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent serious adverse events (SAEs) as of six-week assessment and improvement in key efficacy measures |
| 2023-06-28 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Provides Clinical Updates for Investigational Programs TSHA-120 in Giant Axonal Neuropathy (GAN) and TSHA-102 in Rett Syndrome at R&D Day tayshagtx.com ↗ |
| 2023-05-19 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Presents Preclinical Data on TSHA-102 for Rett Syndrome Demonstrating Cellular Regulation of MeCP2 Expression in Key Mouse Models at the American Society of Gene and Cell Therapy 26th Annual Meeting tayshagtx.com ↗
New preclinical data after neonatal administration in wild-type mice showed no detectable impact on survival, neurobehavioral functions and overall health, suggesting TSHA-102, engineered with novel miRARE technology, avoided toxic overexpression of MeCP2 within cells already expressing MeCP2 |
| 2022-03-29 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-102 in Rett Syndrome tayshagtx.com ↗
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the initiation of clinical development of TSHA-102 for the treatment of Rett syndrome under a recently approved Clinical Trial Application (CTA) by Health Canada. |
| 2021-09-22 | Taysha Gene Therapies, Inc. | Regulatory Taysha Receives Orphan Drug Designation from the European Commission for TSHA-102 for the Treatment of Rett Syndrome tayshagtx.com ↗ |
| 2021-05-11 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Reports First Quarter 2021 Financial Results and Provides a Corporate Update tayshagtx.com ↗
Treatment with TSHA-102 resulted in a statistically significant survival extension by 56% in 4-5 week-old knockout Rett mice with meaningful accumulated disease, a more translatable model of the disorder in humans |
| 2021-05-10 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Announces Publication of Preclinical Data for TSHA-102 in Rett Syndrome in Brain, a Highly Esteemed Neurological Science Peer-Reviewed Journal tayshagtx.com ↗
TSHA-102 resulted in a statistically significant survival extension by 56% in 4-5-week-old knockout Rett mice with meaningful accumulated disease whereas unregulated constructs did not extend survival significantly in the validated MECP2 knockout Rett mouse model |
| 2020-10-27 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-104 to Treat SURF1-Associated Leigh Syndrome tayshagtx.com ↗
Rare pediatric disease and orphan drug designations now obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome and TSHA-118 for CLN1 |
| 2020-10-14 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome tayshagtx.com ↗ |
All press releases naming this drug 36 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | LONIMECGENE RENPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068131 ↗ |
| Known as | Lonimecgen renparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068131 ↗ |
| Known as | TSHA-102 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05606614 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “TSHA-102 is a recombinant, non-replicating, self-complementary AAV9 (scAAV9) vector encoding for the miniMECP2 gene.” NCT05606614 ↗ |
| Route | Intrathecal | “single intrathecal (IT) administration of TSHA-102” NCT05606614 ↗ |
| Target | MECP2 | “TSHA-102 is designed to target the genetic root cause of Rett syndrome by regulating the expression of MECP2 in cells.” NCT05606614 ↗ |