Gene therapy for the central nervous system has been initiated. This expansion will require some degree of simplicity in delivery processes
Moataz Dowaidar
Abstract
In the new development paradigm, gene therapy for the central nervous system has been initiated as a result of the recent successes in phase I–II clinical trials of human patients, as well as superior vector technology. These treatment strategies are no longer confined to diseases with hereditary components, but may now be utilized for a wider array of disease indications and pathways, considerably broadening the scope of gene therapy for the central nervous system (CNS) indications. This expansion will require some degree of simplicity in delivery processes as well as foresight into future IV production requirements, particularly for target-specific delivery. Because of the need for consistent delivery procedures to serve an increasing number of patients, universal delivery procedures which may be used in different locations across the world are necessary. Moreover, manufacturability is critical in the development of the GT sector, as it has a direct impact on the supply of high-quality, large-quantity vectors that are required to meet future clinical demand, as well as the price and ability to introduce novel gene therapy products to the general population.