Myrtelle Inc., has announced a significant leap forward in bringing its first-in-class gene therapy for Canavan disease (CD) to market: the official launch of commercial-stage manufacturing for its product, MYR-101. This critical step marks a transition from development to large-scale production, moving the innovative treatment closer to the patients who desperately need it.

This ambitious manufacturing program is a collaborative effort with two industry giants in gene therapy infrastructure: Charles River and Viralgen Vector Core. Charles River is responsible for the crucial GMP plasmid manufacturing and analytical excellence at its facility in Keele, UK. Simultaneously, Viralgen, a subsidiary of AskBio Inc., is handling the cutting-edge GMP vector production at its world-class facility in San Sebastián, Spain. This strategic partnership combines Myrtelle's proprietary scientific advancements with the robust manufacturing capabilities and quality control of its partners, ensuring that the therapy can be produced with the precision, scalability, and speed required for broad patient access.

At the core of this initiative is MYR-101 (rAAV-Olig001-ASPA), Myrtelle’s groundbreaking gene therapy. What makes MYR-101 unique is its specific tropism for oligodendrocytes, the very cells responsible for producing myelin, which are compromised in Canavan disease. By directly addressing the genetic root of the disorder, MYR-101 aims to restore ASPA enzyme function and support normal brain development. This targeted approach holds the potential to not only halt the progression of this devastating disease but also to facilitate genuine neural repair through remyelination.

Myrtelle's progress has garnered significant recognition from global regulatory bodies. The company's program was notably selected as one of only four participants in the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program, an initiative designed to accelerate the development of treatments for serious rare diseases through enhanced regulatory communication. MYR-101 has also received a comprehensive suite of important designations, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the U.S. FDA. Internationally, it has been granted Orphan Drug Designation and ATMP classification from the European Medicines Agency, as well as Innovative Licensing and Access Pathway (ILAP) status from the UK MHRA. These designations underscore the urgent unmet medical need and the promising potential of MYR-101.

With commercial manufacturing now underway, Myrtelle is rapidly progressing toward finalizing regulatory filings and preparing for commercial launch, pending necessary approvals. This pivotal advancement positions Myrtelle to potentially deliver the first approved therapy for Canavan disease, marking a new chapter in the treatment of rare neurodegenerative conditions and offering a profound sense of hope to patients and their families worldwide.

Canavan disease is a fatal childhood genetic brain disorder caused by mutations in the ASPA gene, which leads to a deficiency of the aspartoacylase enzyme. This deficiency severely impacts brain bioenergetics and development, including the vital process of myelin production. Children with CD typically appear normal for the first few months but then begin to develop severe symptoms such as poor head control, an abnormally large head, difficulty with eye tracking, irritability, diminished muscle tone, and developmental delays. As the disease progresses, seizures, spasticity, swallowing difficulties, and muscle deterioration emerge, often leading to life-threatening complications by approximately 10 years of age. Currently, only palliative treatments are available, making Myrtelle's gene therapy a beacon of hope for this devastating condition.