The Food and Drug Administration (FDA) has granted Fast Track, Rare Pediatric Disease, and Orphan Drug designations to the adeno-associated virus (AAV) vector gene therapy candidate, rAAV-Olig001-ASPA, for the treatment of patients with Canavan disease.

Canavan disease is a rare neurologic disorder characterized by spongy degeneration of the white matter in the brain. The disease is caused by mutations in the gene encoding for aspartoacylase (ASPA), an enzyme produced in oligodendrocytes that breaks down the neurochemical N-acetylaspartate (NAA). Accumulation of NAA in the brain negatively affects myelin production resulting in neurodegeneration.

The AAV gene therapy candidate is designed to deliver the ASPA gene directly to the oligodendrocyte cells in the fluid surrounding the brain and spinal cord. The Company believes that rAAV-Olig001-ASPA can restore ASPA function, thereby reducing concentrations of NAA, and allowing for myelin production. 

The Company is currently evaluating a single dose of rAAV-Olig001-ASPA intracerebroventricularly in up to 24 children with typical Canavan disease in the first-in-human phase 1/2 trial ( Identifier: NCT04833907).

“FDA’s decision to grant these designations for our investigational gene therapy utilizing rAAV-Olig001-ASPA aligns with our mission to provide treatments for patients where few if any options exist and highlights the urgency of developing a treatment for patients with Canavan disease, a devastating disease of young children which results in short life expectancy,” said Nancy Barone Kribbs, PhD, Senior Vice President of Regulatory Affairs at Myrtelle Inc.


  1. Myrtelle receives FDA Fast Track, Rare Pediatric Disease, and Orphan Drug Designations for its proprietary gene therapy for the treatment of Canavan disease. News release. Myrtelle Inc. Accessed March 15, 2022.
  2. Technology. Myrtelle Inc. Accessed March 15, 2022.