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 a vector, called rAAV-Olig001-ASPA to deliver a therapeutic ASPA gene directly to the oligodendrocyte cells that are responsible for producing myelin 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).Clinical Trial
Our company holds global patent families across a range of areas, including unique vector and gene sequences, routes of administration and methods of use
We have built a substantial group of strategic partners including: Pfizer, Rowan University, Dayton Children’s Hospital, amongst others.