Myrtelle teams with Charles River, Viralgen on commercial manufacturing of gene therapy

New York–based Myrtelle Inc., a gene therapy developer focused on neurodegenerative diseases, announced it has initiated commercial-stage manufacturing of MYR-101, its investigational gene therapy for Canavan disease.

Myrtelle is collaborating with Charles River Laboratories and Viralgen Vector Core to produce MYR-101 at commercial scale. Charles River is providing GMP plasmid manufacturing and analytical services at its Keele, U.K. site, while Viralgen, a subsidiary of AskBio, is leading GMP vector production at its facility in San Sebastián, Spain.

The gene therapy, MYR-101 (rAAV-Olig001-ASPA), is reportedly designed with a unique oligodendrocyte-targeting vector that aims to restore ASPA enzyme function, addressing the underlying cause of Canavan disease, a rare genetic neurological disorder characterized by the spongy degeneration of the white matter in the brain. The therapy is intended to support normal brain development by promoting remyelination.

Myrtelle said it is preparing for commercial launch, pending regulatory approvals. The company’s program has received multiple regulatory designations, including RMAT, Orphan Drug, Rare Pediatric Disease, and Fast Track from the U.S. Food and Drug Administration. It has also secured Orphan Drug and ATMP status from the European Medicines Agency, and ILAP designation from the UK MHRA.

MYR-101 is also part of the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program, which is designed to enhance regulatory communication and support for rare disease drug development.

The company said the current manufacturing scale-up is intended to meet anticipated demand if the therapy is approved.