Drug Profile

PBGENE ALS

Alternative Names: PBGENE-ALS

Latest Information Update: 13 Nov 2025

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At a glance

Originator Precision Biosciences
Class Gene therapies
Mechanism of Action C9orf72 protein expression modulators; 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

13 Oct 2025 Preclinical trials in Amyotrophic lateral sclerosis in USA (Parenteral). prior to October 2025 (Precision Biosciences pipeline, October 2025)
11 May 2025 Pharmacodynamics data from a preclinical trial in Amyotrophic lateral sclerosis presented at theAmerican Society of Gene & Cell Therapy (ASGCT-2025)

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