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Myrtelle's Gene Therapy Candidate, rAAV-Olig001-ASPA, Receives RMAT Designation from FDA for Canavan Disease

Wednesday, April 03, 2024

Myrtelle Inc., a clinical-stage gene therapy company focusing on developing transformative treatments for neurodegenerative diseases, has recently announced a significant development. The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation to the company's leading gene therapy product candidate, rAAV-Olig001-ASPA, intended for the treatment of Canavan disease (CD). The RMAT designation program aims to expedite drug development and review processes for promising pipeline drugs, including gene therapies.

Nancy Barone Kribbs, PhD, Senior Vice President of Global Regulatory Affairs at Myrtelle, highlighted the importance of this designation for children affected by Canavan disease. She emphasized that the RMAT program recognizes the potential of this treatment to address the significant unmet medical needs of CD patients who currently lack approved treatment options.

Myrtelle's First-in-Human (FIH) trial utilizes the company's proprietary rAAV vector to directly target oligodendrocytes, the brain cells affected in CD that are responsible for producing myelin—the insulating material essential for proper neuronal function. Canavan disease disrupts normal brain development due to a mutation in the ASPA gene, impairing the production of the enzyme aspartoacylase crucial for brain bioenergetics and myelin production. The gene therapy aims to restore ASPA function and promote healthy brain development in CD patients.

In addition to RMAT designation, rAAV-Olig001-ASPA has received Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA, Orphan Drug Designation & Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency, as well as Innovative Licensing and Access Pathway (ILAP) from the United Kingdom Medicines & Healthcare products Regulatory Agency.



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