CAR-T Safety and Sickle Cell Disease
Two big themes are set to dominate this year. The first is safety. The FDA’s investigation of T-cell malignancies associated with CAR-T products approved for blood cancers may make these treatments less appealing. However, many patients with autoimmune diseases and cancer already accept some risk of secondary malignancies, so this isn’t necessarily going to derail the whole CAR-T space. We can expect plenty of discussion of the likely impact of the news in the panel sessions – and the networking gigs – throughout the ASH weekend.
The other hot topic will be the latest data in sickle cell disease, sparked by the go-ahead for Vertex’s gene editing therapy in the UK. The US is expected to follow with approved very soon, but questions remain about the very small number of patients who can truly benefit from this sophisticated – but expensive – therapy. Expect to hear many views on how we balance these new treatments with existing, more accessible, therapies and how we track the long-term risk of these technologies.
As ever, ASH will see the latest data reveals on CAR-T products, multiple myeloma, leukaemia and more. In this eBook, we outline five key players - Arcellx, Gilead, Regeneron, Vertex and MorphoSys - with a snapshot of their pipelines, commercial outlook, and the data they’re set to present at the conference.