Myrtelle Inc. has announced promising interim results from its Phase 1/2 clinical trial of the gene therapy candidate rAAV-Olig001-ASPA (MYR-101) for Canavan disease, a devastating neurodegenerative disorder.

Canavan disease is a fatal genetic disorder affecting children, caused by ASPA mutations that lead to severe neurodevelopmental decline, with no current cure and only palliative treatments available.

The ongoing study involved eight children with typical Canavan disease, followed for up to two years, all of whom showed developmental improvements across multiple assessments, indicating broad functional gains.

Myrtelle’s innovative approach uses a proprietary recombinant AAV vector to deliver the ASPA gene directly to oligodendrocytes, aiming to restore enzyme activity, improve NAA metabolism, and promote myelin repair.

The therapy targets oligodendrocytes to produce aspartoacylase, the enzyme deficient in Canavan disease due to ASPA mutations, which impairs myelination in the brain.

The therapy has received multiple regulatory designations, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track from the FDA, along with similar designations from EMA and MHRA, supporting its potential for expedited development.

Myrtelle has an exclusive licensing agreement with Pfizer and is actively advancing this gene therapy through clinical development, with ongoing follow-up to evaluate long-term outcomes.

Results from the trial indicate that MYR-101 was well-tolerated, with no serious adverse events, and showed significant reductions in cerebrospinal fluid NAA levels and increases in brain myelin volume, suggesting effective disease modification.