Accelerating gene therapy development: from concept to clinic

Cell & Gene Therapy Insights 2021; 7(12), 1807–1824


Published: 20 January 2022
Innovator Insight
Steven Gill, Qian Lui, Ryan Cawood

Adeno-associated viral (AAV) vectors remain the tool of choice for today’s gene therapy manufacturers – but as demand increases, the limitations of traditional manufacturing methods may severely impact progress of the field. This article discusses a case study on the development of a novel gene therapy for motor neuron disease (MND) and Parkinson’s disease (PD), before discussing how next generation technologies can help overcome manufacturing challenges and accelerate the development of novel gene therapies.