Infantile GM2 Gangliosidosis (Disorder)

1
Pipeline Programs
1
Companies
1
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Taysha Gene Therapies
1 program
1
TSHA-101Phase 1/21 trial
Active Trials
NCT04798235Active Not Recruiting3Est. Mar 2027

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
Taysha Gene TherapiesTSHA-101

Clinical Trials (1)

Total enrollment: 3 patients across 1 trials

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

Start: Mar 2021Est. completion: Mar 20273 patients
Phase 1/2Active Not Recruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 companies competing in this space