Myrtelle 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.
"This is a transformative moment for Myrtelle and the entire Canavan disease community," said Adrian Stecyk, Chief Executive Officer of Myrtelle. "Initiating commercial manufacturing represents the culmination of our bold vision—to translate deep scientific innovation into real-world therapies. With Charles River and Viralgen as our partners, we are poised to deliver a life-changing treatment with precision, scalability, and speed."
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.
"When every day, every hour, every minute makes a difference in saving lives, the Charles River team is excited and privileged to continue to support Myrtelle at this pivotal stage," said Kerstin Dolph, Corporate Senior Vice President of Global Manufacturing at Charles River. "Myrtelle's groundbreaking program exemplifies the progress gene therapy has made from concept toward commercial reality, and we're proud to be part of that journey, bringing hope to the patients and families affected by Canavan disease."
"Our mission at Viralgen is to transform people's lives through our manufacturing science," said Jimmy Vanhove, Chief Executive Officer at Viralgen. "Myrtelle's work in Canavan disease is extraordinary, and we are honored to contribute our expertise and advance GMP production to help make this therapy a reality."