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# Myrtelle Gets 3 FDA Designations for Gene Therapy
- URL: https://www.fdaweb.com/myrtelle-gets-3-fda-designations-for-gene-therapy/
- Published: 2022-03-17T12:00:00.000Z
- Updated: 2026-09-14T17:36:54.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5151395

FDA has granted Myrtelle a Fast Track, Rare Pediatric Disease, and Orphan Drug designations for its lead clinical-stage gene therapy, rAAV-Olig001-ASPA, for treating patients with Canavan Disease. The condition is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent normal Aspartoacylase enzyme expression, a “critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA),” the company says. “When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health.”  

An ongoing Phase 1/2 clinical trial utilizes a novel proprietary recombinant adeno-associated virus vector, AAV-Olig, that the company says for the first time directly targets oligodendrocytes in the brain. The oligodendrocyte-targeted gene therapy is intended to restore ASPA function, enable metabolism of the abundant brain chemical NAA, and support myelination.  

Myrtelle entered into a licensing agreement with Pfizer Inc. in 2021 to develop and commercialize the gene therapy.