Vertex has become a biotech investor favorite amid the tumult of 2020, but the potential of its pipeline is now under question.

With gene editing making the news again after the recent Nobel Prize for CRISPR, China’s EdiGene has been raising new funds to accelerate its pipeline to the clinic, led by a potential one-time cure for thalassemia and helped by recent regulatory changes.

Among the six new investigational products to make it onto the European Medicines Agency’s priority medicines scheme, five were gene therapies.

With gene therapies for sickle cell disease looming and two new expensive therapies of to a slow start, access challenges could prevent a large portion of patients from receiving treatment, a report from the US National Academies of Sciences, Engineering and Medicine concludes. The report urges development of new payment models, offering five ideas.