Myrtelle Inc. is presenting a company overview and encouraging interim results from its phase 1/2 clinical trial of the investigational gene therapy rAAV-Olig001-ASPA (MYR-101) for Canavan disease.
The presentation is being given at the 2025 Cell & Gene Meeting on the Mesa, organized by the Alliance for Regenerative Medicine (ARM) in Phoenix, Arizona.
It marks an important milestone in Myrtelle’s mission to bring the first disease-modifying therapy to children living with Canavan disease—a devastating, fatal leukodystrophy caused by a single-gene defect. The results highlight the successful clinical translation of Myrtelle’s oligodendrocyte-targeted AAV platform, designed to address the underlying cause of the disease by restoring myelin-forming cell function in the brain.
Key findings from the study were recently published in Nature Medicine. MYR-101 demonstrated a favourable safety profile with no serious adverse events related to treatment. There were significant reductions in N-acetylaspartate (NAA) levels in cerebrospinal fluid (CSF), consistent with restoration of ASPA enzyme function. Increases in brain myelin volume measured by Synthetic MRI (SyMRI) confirm remyelination consistent with therapeutic benefit.
Participants also demonstrated measurable developmental progress compared with historical controls, reflecting broad functional gains, while an ongoing follow-up continues to show promising long-term outcomes and durability of effect.
“These data represent a major step forward for children and families affected by Canavan disease,” said Michael Muhonen, co-chief medical officer at Myrtelle.
“For the first time, we’re seeing clear biological and functional evidence that gene therapy targeting oligodendrocytes can directly address the core pathology of this disease.”
rAAV-Olig001-ASPA (MYR-101) was selected by the U.S. Food and Drug Administration (FDA) for inclusion in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot programme, one of only four gene therapies regulated by CBER to receive this distinction. START provides enhanced FDA engagement to accelerate the development of promising therapies for rare diseases.
Jim Cornall is editor of Deeptech Digest and publisher at Ayr Coastal Media. He is an award-winning writer, editor, photographer, broadcaster, designer and author. Contact Jim here.
