The National Institute of Health (NIH) has launched a $190 million program to advance the use and development of gene therapies to clinic. The six year project will distribute the fund amongst studies that are considered high impact.
The focus of the program will be on the gene editing of somatic cells; this eliminates the possibility of DNA alterations being inherited. Current practices involve the ex situ editing of cells using CRISPR, but the ultimate aim is to edit genes in situ. Examples of ongoing, or shortly commencing studies at present include the delivery of edited T cells to cancer patients by the University of Pennsylvania, and the European trialling of gene editing in beta-thalassemia patients that is being carried out by Vertex and CRISPR in collaboration.
The NIH project hopes to build a toolkit of sorts for researchers in the area by focusing on improving areas such as tissue targeting and editing techniques.
NIH Director Francis Collins commented that the aim of the investment is to ‘dramatically accelerate the translation of these technologies to the clinic for treatment of as many genetic diseases as possible.’ This is particularly apt given the number of regulartory barriers to market in the US, which is contrasted by the rapid development of comparable technologies in China.
Source: NIH to plow $190M into genome editing therapy drive Press Release