The upcoming review of two novel gene therapies from bluebird bio marks will be the first time the US FDA’s CTGTAC has met for a genetic product review in five years. That is a surprisingly long gap – and one that probably will not be repeated.

Draft from US FDA advises sponsors on how to draw from platform knowledge, while anticipating issues associated with emerging centralized and distributed manufacturing approaches for autologous and allogeneic products and their viral vectors.

CEO of Japanese ophthalmology specialist lays out clear-sighted business vision as track laid for expansion into global markets and new indications amid expiry challenges at home. New modalities are also being explored to meet needs in demanding disorders, Shigeo Taniuchi tells Scrip in an exclusive interview. 

Plagued by a scandal which gained global attention a few years go, the gene therapy sector has recovered and is now thriving in China. But a drive for innovation, combined with ready cash for aspiring start-ups, may not translate into leaps and bounds in the field due to challenges unique to these latest treatments, including manufacturing, durability and coverage.