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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
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