FDA, Others Working on Rare Disease Gene Therapies

FDA, the National Institutes of Health (NIH), 10 drug companies, and five non-profit organizations are partnering to accelerate the development of gene therapies for the 30 million Americans who suffer from rare diseases. An NIH statement says the new Bespoke Gene Therapy Consortium “aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with rare diseases.”

A primary aim of the consortium is to improve understanding of the basic biology of a common gene delivery vector known as the adeno-associated virus (AAV), NIH says. “Researchers will examine the biological and mechanistic steps involved in AAV vector production, vector delivery of genes into human cells, and how therapeutic genes are activated in target cells, NIH says. “Such tests could be broadly applicable to different manufacturing methods and make the process of developing gene therapies for very rare conditions much more efficient,” it says.