Gene therapy for the treatment of spinal muscular atrophy

The FDA has now approved three innovative gene targeting treatments for the treatment of individuals with spinal muscular atrophy (SMA), each with its own unique delivery method. The need for therapy optimization is underlined by the variety in clinical outcomes, notwithstanding this remarkable success. More basic and translational research is needed to determine the factors that limit cell and tissue medication biodistribution and target engagement, as well as to assess long-term durability and potential toxicities. This research in SMA will be crucial in the development of successful gene-targeting therapeutics for other neurogenetic illnesses. The expensive expense of SMA therapies has received a lot of media attention. Nusinersen costs $125,000 each dose, with numerous treatments costing more than $1 million; while risdiplam is scheduled to be price-capped at up to $340,000 each year (91, 92). The cost of gene-targeting drugs is unsustainable, especially because the US Food and Drug Administration anticipates approving 10 to 20 cell or gene therapy products each year by 2025. Efforts must be taken to address these rising costs in order for all patients to benefit from precision medicine.