Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D1TS4V
|
|||
| Drug Name |
TSHA-101
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | GM2 gangliosidosis [ICD-11: 5C56.00] | Phase 1/2 | [1] | |
| Company |
Taysha Gene Therapies Dallas, TX
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Beta-hexosaminidase subunit alpha (HEXA) | Target Info | . | [1] |
| Beta-hexosaminidase subunit beta (HEXB) | Target Info | . | [1] | |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04798235) Phase 1/2, Open-Label Clinical Study to Evaluate the Safety and Efficacy of Intrathecal TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis. U.S.National Institutes of Health. | |||
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