Most recently approved gene therapy products use an adeno-associated virus (AAV) as their gene delivery vector. For example, Elevidys, a new treatment for paediatric patients with DMD that was ...

“We are building world-class capabilities to discover, develop, manufacture, and commercialize gene-therapy products. Our neuroanatomy-led approach to drug delivery should have utility across ...

"We resolved critical bottlenecks and achieved a much more efficient packaging by the delivery agents." Advancing Cancer Therapy ... gene editors within the production cells. By adjusting ...

Over the past decade the application of gene therapy of ... of administration of the delivery vector and can include nonspecific binding and cellular association. Intracellular barriers are ...

2 However, not all vectors have equivalent properties, with some possessing higher packaging capacities, delivery ... therapy. To ensure they have the materials required for each testing phase, ...