In spite of positive results yielded from dozens of gene therapy trials, to date neither Europe nor the US has given the go ahead for routine use of a single form of gene therapy in the clinic.
All this may be about to change as calls, and actions, to give patients “the treatments they deserve” gain momentum. A commentary in Human Genome Therapy by Dr James Wilson, argues that early indications of gene therapy as a viable treatment for haemophilia B, and potentially haemophilia A, indicate the time is ripe for the commercial development of such therapies.
However, he acknowledges the caution of commercial developers of these disruptive technologies who need to find new business models that will sustain them in what are, at least for the time being, niche markets.
One biotechnology organisation that is ramping up its activity in the field is French not-for-profit, Genethon. The institute has recently taken the decision to manufacture clinical-grade viral vectors. Access to vectors is currently a major bottleneck in moving gene therapies into the clinic. Genethon’s new Scientific Director, Fulvio Mavilio says the aim is to make the institute “a major hub for gene therapy networks”.