Drug Profile

GS 001

Alternative Names: GS-001; GS-1191

Latest Information Update: 06 May 2025

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At a glance

Originator Gritgen Therapeutics
Class Antihaemorrhagics; Gene therapies
Mechanism of Action Factor VIII replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

Highest Development Phases

Phase III Haemophilia A

Most Recent Events

31 Mar 2025 Phase-III clinical trials in Haemophilia A in China (IV) (NCT06833983)
19 Feb 2025 Gritgen Therapeutics plans a phase III trial for Hemophilia A in China (IV) (NCT06833983)
07 Dec 2024 Updated efficacy, immunogenicity and adverse events data from a phase I trial in Haemophilia A presented at the 66^th American Society of Hematology Annual Meeting and Exposition (ASH-Hem-2024)

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