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TSHA-101 · 1 trial · 1 indication
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| Arm | Type | Description |
|---|---|---|
| TSHA-101 | EXPERIMENTAL | Subjects who will receive one-time intrathecal TSHA-101, brain volume based sliding scale for dosage |
| Name | Type | Description |
|---|---|---|
| TSHA-101 | BIOLOGICAL | AAV9 viral vector containing HEXA and HEXB genes to be administered via Intrathecal injection |
Key Inclusion Criteria: * male or female with age less than or equal to 15 months * diagnosis of GM2 gangliosidosis with genetic and enzymatic documentation of infantile disease Key Exclusion Criteria: * a second neurodevelopmental disorder independent of the HEXA or HEXB * inability to tolerate ...
TSHA-101 is an investigational gene therapy being developed for the treatment of infantile GM2 gangliosidosis, a rare inherited disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
TSHA-101 targets the HEXA and HEXB genes, which are involved in the production of enzymes needed to break down GM2 gangliosides. By delivering functional copies of these genes, the therapy aims to address the underlying genetic cause of infantile GM2 gangliosidosis.
TSHA-101 is being developed by Taysha Gene Therapies, Inc., a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy.
TSHA-101 is in Phase 1 clinical development. It is an investigational drug and has not yet received FDA approval. The ongoing Phase 1 trial is a first-in-human study designed to assess the therapy's safety and preliminary efficacy.
TSHA-101 is being studied in a Phase 1 clinical trial with the identifier NCT04798235. This first-in-human study is evaluating the gene therapy for the treatment of infantile onset GM2 gangliosidosis. The trial is active but not recruiting participants.
TSHA-101 is a distinct investigational gene therapy developed by Taysha Gene Therapies. It is not known to be marketed under any other names. Its unique mechanism targets the HEXA and HEXB genes for the treatment of infantile GM2 gangliosidosis.