26 Jul Myrtelle
Michael Muhonen, M.D., Chief Medical Officer
Oct. 7 | 4:00pm | FLW Ballroom G
New York, NY
(Private)
Myrtelle is a patient-centered, innovation-driven gene therapy company transforming the treatment of neurological diseases. Our mission is to develop and deliver novel treatments for devastating myelin-based disorders of the central nervous system for which few, if any, treatment options exist. Myrtelle’s clinical program for Canavan disease is the first gene therapy to specifically target oligodendrocytes. rAAV-Olig001-ASPA has been selected for FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program and granted Orphan Drug, Fast-Track and Rare Pediatric Disease Designations for the treatment of Canavan disease in the US, with similar designations in Europe. Myrtelle recently announced published interim results of its Phase 1/2 Clinical Trial of rAAV-Olig001-ASPA for Canavan disease in Nature Medicine reporting the investigational oligodendrocyte targeted drug leads to decreased N-acetylaspartate (NAA) levels and increased brain myelin volume, resulting in promising functional improvements in children with Canavan disease.
