Drug Profile

AS 241

Alternative Names: AS-241; UNC13A targeting antisense oligonuclotide therapeutic - AcuraStem

Latest Information Update: 11 May 2026

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At a glance

Orphan Drug Status

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

No
New Molecular Entity Yes

28 Apr 2026 Preclinical trials in Amyotrophic lateral sclerosis is underway in USA (Parenteral)
28 Apr 2026 Preclinical trials in Frontotemporal dementia is underway in USA (Parenteral)
28 Apr 2026 AcuraStem plans a phase I trial for Frontotemporal dementia and Amyotrophic lateral sclerosis (Parenteral)

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