Drug Profile

ART 001 - AccurEdit Therapeutics

Alternative Names: ART-001 - AccurEdit Therapeutics

Latest Information Update: 31 Jul 2025

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At a glance

Originator AccurEdit Therapeutics
Class Gene therapies
Mechanism of Action Gene modulators; Prealbumin expression inhibitors

Orphan Drug Status

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

Yes - Transthyretin-related hereditary amyloidosis
New Molecular Entity No

Highest Development Phases

Phase I/II Transthyretin-related hereditary amyloidosis

Most Recent Events

29 May 2025 ART 001 receives Regenerative Medicine Advanced Therapy (RMAT) status for Transthyretin-related hereditary amyloidosis in USA
12 May 2025 Phase-I clinical trials in Transthyretin-related hereditary amyloidosis in USA (IV)
20 Mar 2025 ART 001 receives Orphan Drug status for Transthyretin-related hereditary amyloidosis in USA

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