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.

Data for new genetic methods to tackle severe hemoglobinopathies presented at the EHA meeting bring more confidence for bluebird’s gene therapy LentiGlobin and early suggestions of a functional cure with CRISPR gene editing with CRISPR Therapeutics/Vertex’s CTX001.

BIO hosted discussions during its annual meeting to reinforce the value of inclusion, offer advice to companies and reflect on recent US events.

BIO hosted discussions during its annual meeting to reinforce the value of inclusion, offer advice to companies and reflect on recent US events.