The US biotech has released new positive data from two late-stage studies in the rare disease after the US FDA asked for more controlled data following an initial disappointment.

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.

Gene therapies for conditions ranging from hemophilia to retinal diseases are blossoming in China and the race in which the winner may take all is on. Driven by ready investors, the field is on the cusp of becoming the latest innovation battleground in the country, while some developers are already moving ahead in the US.

Gene therapies for conditions ranging from hemophilia to retinal diseases are blossoming in China and the race in which the winner may take all is on. Driven by ready investors, the field is on the cusp of becoming the latest innovation battleground in the country, while some developers are already moving ahead in the US.