Myrtelle has published interim results from its Phase 1/2 trial of the investigational gene therapy rAAV-Olig001-ASPA (MYR-101) in Nature Medicine, reporting changes in biomarkers, imaging and functional measures in children with Canavan disease, according to the company.
The trial includes eight children with typical Canavan disease, followed for up to two years post-treatment. The company said that MYR-101 was generally well tolerated, with no serious adverse events attributed to the gene therapy itself. It reported a significant reduction in N-acetylaspartate (NAA) levels in cerebrospinal fluid (CSF), increases in brain myelin volume measured by synthetic MRI, and developmental improvements in participants compared with historical controls as assessed by the Mullen Scales of Early Learning (MSEL). All treated children improved in at least two MSEL domains; most improved in three or more domains.
Myrtelle claims that these findings support the potential of MYR-101 as a disease-modifying therapy. The therapy is designed to deliver a functional ASPA gene to oligodendrocytes, addressing the enzymatic deficiency that underlies the disease.
On the regulatory front, the company noted that MYR-101 has received multiple designations — including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, Rare Pediatric Disease, Fast Track in the U.S., and equivalent designations in Europe and the UK. The U.S. Food and Drug Administration has also included MYR-101 in its START pilot program, which provides enhanced regulatory support for rare disease gene therapies.
Myrtelle holds an exclusive worldwide license with Pfizer for its Canavan disease program.
