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# Gene Therapy for ALS Get Fast Track Status
- URL: https://www.fdaweb.com/gene-therapy-for-als-get-fast-track-status/
- Published: 2021-07-28T12:00:00.000Z
- Updated: 2026-09-14T17:08:02.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5149813

FDA has granted Apic Bio a fast track designation for APB-102, a gene therapy candidate designed to treat SOD1 amyotrophic lateral sclerosis (ALS). The company is planning a Phase 1/2 clinical trial in early 2022\. Apic says the three-part study is intended to evaluate the safety, tolerability, and efficacy of intrathecally administered APB-102 in patients with SOD1 ALS mutations: Part I, single ascending dose; Part II, randomized, double-blind, placebo-controlled; and Part III, extended follow-up.  

In ALS, mutations in the SOD1 gene account for about one-fifth of all inherited forms of the disease, the company says. APB-102 is designed to target the underlying pathophysiology of the disease, it says. The therapy is described by Apic as a recombinant AAVrh10 vector that expresses an anti-SOD1 artificial microRNA. “The microRNA binds to SOD1 mRNA thereby reducing production of the mutant protein in patients with this form of the disease,” it says. “Reducing mutant SOD1 protein levels may improve survival and function of motor neurons and potentially provide a therapeutic benefit to people with SOD1-linked ALS.”