Myrtelle Inc. Launches Manufacturing of First-in-Class Gene Therapy for Canavan Disease in Strategic Alliance with Charles River and Viralgen Vector Core

Published: 2025-07-08 12:20:31 ET CRL

Published on 07/08/2025 at 12:20

Myrtelle Inc. announced the official launch of commercial-stage manufacturing for its first-in-class oligotrophic recombinant adeno-associated virus (rAAV) gene therapy product, developed specifically for Canavan disease (CD). This landmark achievement propels Myrtelle's therapy closer to market and patients who urgently need it. The manufacturing program is being executed in strategic partnership with two global leaders in gene therapy infrastructure: Charles River and Viralgen Vector Core (a subsidiary of AskBio Inc.).

Together, the trio is setting a new standard in viral vector innovation, combining Myrtelle's proprietary science with Charles River's GMP plasmid manufacturing and analytical excellence at their CDMO center of excellence in Keele, UK, and Viralgen's cutting-edge GMP vector production at their world-class facility in San Sebastián, Spain. At the heart of this initiative is MYR-101, Myrtelle's pioneering rAAV-Olig001-ASPA gene therapy. Engineered with a unique tropism for oligodendrocytes?the myelin-producing cells compromised in Canavan disease?MYR-101 is designed to directly address the genetic root of the disorder by restoring ASPA enzyme function and supporting normal brain development.

This targeted approach has the potential not only to halt disease progression but to enable true neural repair through remyelination. Myrtelle's gene therapy program continues to gain momentum on multiple fronts. The Company remains on track to finalize regulatory filings and prepare for commercial launch, pending approvals.

MYR-101 was selected as one of just four programs to participate in the FDA'sSupport for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program, a groundbreaking initiative to accelerate development of treatments for serious rare diseases by enhancing regulatory communication and flexibility. The company's progress has also been recognized globally with multiple key designations awarded to rAAV-Olig001-ASPA, including: RMAT, Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the U.S. FDA; Orphan Drug Designation and ATMP classification from the European Medicines Agency; Innovative Licensing and Access Pathway (ILAP) status from the UK MHRA. With manufacturing now underway in preparation for commercialization, Myrtelle is accelerating toward its goal of delivering the first approved therapy for Canavan disease, opening a new chapter in the treatment of rare neurodegenerative conditions?and offering a profound message of hope to patients and families around the world.