TSHA-101

Biologic

DDr. Anupam Sehgal

TSHA-101 is one of 6 biologic programs targeting GM2 gangliosidosis. It is currently in development.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

GM2 GangliosidosisActive, not recruiting

GM2 GangliosidosisPhase 1/Phase 2

Active, not recruiting

Competition

Competitive Landscape

6 competitors in GM2 Gangliosidosis

#DrugCompanyPhaseTrials

Opfolda

Johnson & Johnson

Phase 2

1 2

Daraprim

FFondazione Matilde Tettamanti Menotti De Marchi Onlus

Phase 1

1 3

IB1001

MMedexus Pharma, Inc.

Phase 2

1 4

Wellcovorin

EEastern Cooperative Oncology Group

AZ-3102

2 6

TSHA-101

DDr. Anupam Sehgal

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2027

Phase 1/2Active, not recruiting

GM2 Gangliosidosis

NCT04798235

Clinical

Trials by Indication[1]

GM2 gangliosidosis1 trial

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

Active, not recruiting·—·Phase 1/2·NCT04798235·3 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for TSHA-101. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — TSHA-101 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for TSHA-101. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Dr. Anupam Sehgal company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Biologic[2]
Company: DDr. Anupam Sehgal

TSHA-101

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Trials by Indication[1]

GM2 gangliosidosis1 trial

References

Drug Info

Quick Facts

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