Myrtelle is advancing proprietary technologies to optimize the delivery of genes to myelin-generating cells, enabling innovative approaches to address the needs of hundreds of disorders involving myelin.


Myrtelle is developing gene therapy vectors to directly target oligodendrocyte cells responsible for producing myelin. The vector used in our lead Canavan program called rAAV-Olig001-ASPA is intended to restore the ability of oligodendrocytes to make the ASPA enzyme on their own.

The ASPA gene is delivered directly into the fluid that surrounds the brain and spinal cord (cerebrospinal fluid), allowing the gene therapy to reach many cells. The gene is packaged using a unique adeno-associated virus (AAV) vector that targets oligodendrocytes. The virus is inactive and cannot cause disease.  Once the ASPA gene is inside the cells, researchers believe it may help the cells make and release Aspartoacylase (ASPA), the enzyme needed to breakdown N-Acetylaspartate (NAA).

See Our Current Trials

Intellectual Property

Our company holds global patent families across a range of areas, including unique vector and gene sequences, routes of administration, and methods of use.


Collaborations that Lead to Change

We have built a substantial group of strategic partners including Pfizer, Rowan University, and Dayton Children’s Hospital, among others.


Understanding Gene Therapy for Rare CNS Diseases