Gene Therapy Halts Progression of Rare Genetic Condition in 4-yr Old Toronto Boy Michael

Jun 28, 2024, 11:33 PM

A groundbreaking gene therapy has halted the progression of a rare genetic condition in a young boy from Toronto. The therapy, conducted at SickKids Hospital and published in Nature Medicine, involved a personalized AAV9-based gene therapy for hereditary spastic paraplegia type 50. The phase 1 trial, which included only one patient, showed that the therapy was well tolerated and led to preliminary disease stabilization within three years from diagnosis. The patient, Michael, a 4-yr old, is the son of Terry Pirovolakis, who played a significant role in making this treatment possible.