Myrtelle

About Myrtelle

Myrtelle Inc. is a clinical-stage gene therapy company, founded in 2021 and headquartered in Wakefield, Massachusetts, that concentrates on creating treatments for neurodegenerative diseases. The company's business model is centered on the development and commercialization of a proprietary platform of gene therapies aimed at treating myelin-based disorders. Myrtelle's financial strategy involves securing funding through various rounds, including debt financing, to advance its clinical trials and research programs.

The company was co-founded by Adrian Stecyk, who now serves as the Chief Executive Officer. Stecyk's background includes co-founding several other gene therapy companies, such as Agilis Biotherapeutics, and an early career at Draper Labs, an R&D company focused on advanced technological solutions. His experience is complemented by his work at Griffin Securities, an investment bank that supports emerging biotechnology companies. This journey reflects a long-standing commitment to advancing biotechnological research and development.

Myrtelle's primary focus is on rare central nervous system diseases with significant unmet medical needs. Their lead program targets Canavan disease, a fatal childhood genetic brain disorder. This is addressed through their primary product, MYR-101 (also known as rAAV-Olig001-ASPA), an investigational gene therapy. This therapy is distinctive as it's the first to specifically target oligodendrocytes, the brain cells responsible for producing myelin. The therapy utilizes a recombinant adeno-associated virus (rAAV) vector to deliver a functional copy of the ASPA gene, which is mutated in Canavan disease. The goal is to restore the function of the ASPA enzyme, reduce the buildup of the neurochemical N-Acetylaspartate (NAA), and consequently restore brain development and myelination. The therapy is administered as a one-time dose into the brain's lateral ventricles.

Beyond Canavan disease, Myrtelle's pipeline includes programs for other white matter disorders such as Pelizaeus-Merzbacher Disease (PMD), Multiple System Atrophy (MSA), H-ABC, and a form of monogenic hearing loss (DFNB8). The company has received multiple regulatory designations from the FDA for its Canavan disease program, including Fast Track, Rare Pediatric Disease, and Orphan Drug designations, which are intended to expedite the development and review process. Myrtelle also holds an exclusive worldwide licensing agreement with Pfizer for its Canavan disease program.