- Myrtelle has launched commercial manufacturing for its gene therapy targeting Canavan disease, in partnership with CDMOs Charles River and Viralgen.
- The therapy, MYR-101, is designed to restore ASPA enzyme function in oligodendrocytes and is supported by regulatory designations in the US, UK, and EU.
Myrtelle Inc. has commenced commercial-stage manufacturing for its investigational gene therapy product, MYR-101, developed for the treatment of Canavan disease. The initiative marks a key milestone in the company’s journey toward market readiness and expanded patient access.
The manufacturing programme is being carried out in collaboration with two CDMO partners: Charles River, which will provide GMP plasmid manufacturing and analytics from its facility in Keele, UK, and Viralgen, which will oversee GMP vector production in San Sebastián, Spain. The partnership aims to combine scientific innovation with scalable, regulatory-compliant production capabilities.
“This is a transformative moment for Myrtelle and the entire Canavan disease community,” said Adrian Stecyk, CEO of Myrtelle. “With Charles River and Viralgen as our partners, we are poised to deliver a life-changing treatment with precision, scalability, and speed.”
MYR-101 is based on a proprietary recombinant adeno-associated virus (rAAV) vector, rAAV-Olig001-ASPA, which is engineered to target oligodendrocytes. These myelin-producing cells are directly impacted by Canavan disease. The therapy aims to restore ASPA enzyme function and enable remyelination, offering potential to halt disease progression and support neural repair.
The therapy has been selected for the FDA’s START Pilot Program and holds RMAT, Orphan Drug, Rare Paediatric Disease, and Fast Track designations from the US FDA. Additional designations include Orphan Drug and ATMP status from the EMA, and ILAP designation from the UK MHRA.
With manufacturing now in motion, Myrtelle is preparing regulatory filings and advancing toward commercial launch, aiming to deliver the first approved treatment for Canavan disease.
