33 states pick up CMS program to pay for sickle cell gene therapies

States representing about 84% of Medicaid beneficiaries are participating in an initiative that enables the U.S. government to negotiate pay-for-performance deals with the manufacturers of products like Casgevy and Lyfgenia. 

FDA rejects Ultragenyx gene therapy over manufacturing concerns

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather than a roadblock.”

AI and machine learning trends in the pharmaceutical industry

FDA turns back Capricor’s Duchenne cell therapy

The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the agency during deramiocel’s review.

Scientists show that mRNA therapy can be delivered as a capsule

Four autism subtypes map onto distinct genes, traits

Patients Fight for Access to BrainStorm’s ALS Drug After Expanded Access Data ‘Defy Logic’