Remove tag cancer-drugs-fund
article thumbnail

Second unanimous FDA adcomm vote boosts bluebird bio

pharmaphorum

Cases of cancer associated with insertional mutagenesis, caused when the viral vectors used to deliver the gene therapy to host cells, have been seen with bluebird’s gene therapies but have been more apparent in trials involving its CALD and sickle cell disease candidates than beti-cel.

FDA 59
article thumbnail

Oslo Medicines Initiative asks how to balance innovation incentivisation with security of access

pharmaphorum

Flawed funding model. It has become clear that the medicines of the 20 th century, the so-called blockbuster era, are gradually being replaced by drugs designed for smaller groups, specific indication, and even for individual patients,” said Jo De C**k, CEO of Belgium’s National Institute for Health and Disability Insurance.

article thumbnail

Developing point-of-care CAR T manufacturing

European Pharmaceutical Review

Chimeric antigen receptor (CAR) T cells are the first example of a “living drug”. Being a cellular therapy that involves cell manipulation and molecular transfection – and above all, being a living drug – makes CAR T-cell therapy a somewhat challenging immunotherapy to manufacture in‑house. US Food and Drug Administration.