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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
NCTID
NCT04798235
(View at clinicaltrials.gov)
Description
GM2 gangliosidoses are a group of autosomal recessive neurodegenerative diseases characterized by a deficiency of the Hex A enzyme to catabolize GM2, thereby causing GM2 accumulation within cellular lysosomes.Hex A is composed of 2 subunits, α- and β-, coded by the HEXA and HEXB genes, respectively. The primary purpose of the current study is to assess the safety and tolerability of TSHA101 administered via IT injection.
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Development Status
Active
Indication
Infantile GM2 Gangliosidosis
Disease Ontology Term
DOID:3320|DOID:3323
Compound Name
TSHA-101
Sponsor
Dr. Anupam Sehgal
Funder Type
Network
Recruitment Status
Active not recruiting
Enrollment Count
3
Results Posted
Not Available
Therapy Information
Target Gene/Variant
HEXA/HEXB
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intrathecal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV9
Editor Type
Dose 1
5E14 vg (n=3)
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2021-02-22
Completion Date
2027-03-12
Last Update
2023-05-09
Participation Criteria
Eligible Age
<=15 Months
Standard Ages
Child
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
Canada
Regulatory Information
Has US IND
False
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates
The Company announced they had transferred rights back to Queen's University in February 2024
Resources/Links
News and Press Releases
Taysha Gene Therapies Provides Update on Deprioritized Pipeline Programs
SEC Form 10-K: Taysha Gene Therapies, Inc. FY2024
SEC Form 10-K: Taysha Gene Therapies, Inc. FY2021