Drugs / TSHA-101
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04798235 | Mar 2021 → Mar 2027 expected | GM2 gangliosidosis | Dr. Anupam Sehgal | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2022-01-27 | Taysha Gene Therapies, Inc. | Results Taysha Gene Therapies Announces Positive Initial Biomarker Data For TSHA-101, the First Bicistronic Gene Therapy in Clinical Development, Demonstrating Normalization of β-Hexosaminidase A Enzyme Activity in Patients With GM2 Gangliosidosis 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 reported positive initial serum β-hexosaminidase A (Hex A) enzyme activity data for TSHA-101 in patients with Sandhoff and Tay-Sachs diseases, which represent two forms of GM2 gangliosidosis. |
| 2021-09-29 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis 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 that it has been granted orphan drug designation from the European Commission for TSHA-101, an AAV9-based bicistronic gene replacement therapy in development for GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease. |
| 2020-12-21 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Announces Queen’s University’s Receipt of Clinical Trial Application Approval from Health Canada for Phase 1/2 Clinical Trial of TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis tayshagtx.com ↗
today announced that Queen’s University in Ontario, Canada, received Clinical Trial Application (CTA) approval from Health Canada for its investigator-sponsored Phase 1/2 trial exploring TSHA-101, Taysha’s investigational AAV9-based gene therapy, for the treatment of infantile GM2 gangliosidosis. |
| 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 |
| 2020-08-27 | Taysha Gene Therapies, Inc. | Regulatory Taysha Gene Therapies Receives Orphan Drug Designation and Rare Pediatric Disease Designation for TSHA-101 for GM2 Gangliosidosis tayshagtx.com ↗
DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, a patient-centric gene therapy company with a mission to eradicate monogenic CNS diseases, today announced that it has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for TSHA-101, an AAV9-based gene therapy in development for GM2 Gangliosidosis. |
All press releases naming this drug 6 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | TSHA-101 | “First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis” NCT04798235 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “AAV9 viral vector containing HEXA and HEXB genes” NCT04798235 ↗ |
| Route | Intrathecal | “administered via Intrathecal injection” NCT04798235 ↗ |