Drug Profile

RM 004

Alternative Names: RM-004

Latest Information Update: 18 Apr 2025

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At a glance

Originator Reforgene Medicine
Class Gene therapies
Mechanism of Action 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 0 Alpha-thalassaemia

Most Recent Events

07 Dec 2024 Efficacy and adverse event data from phase 0 trial in Alpha thalassaemia presented at the 66th American Society of Hematology Annual Meeting and Exposition (ASH-Hem-2024)

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