US FDA has received a total of 19 reports of T-cell malignancy involving BCMA- or CD19-directed autologous CAR-T cell immunotherapies and is evaluating the need for regulatory action. Focus on lentiviral and retroviral vectors could extend to safety of other types of gene therapies.

AstraZeneca’s Truqap and Evive’s Ryzneuta bring FDA center for drugs’ 2023 novel agent total to 51, while biologics center already sits on a record-breaking 14 novel approvals.

Plus deals involving Abliva/Owl, Selecta/Cartesian, Novo Nordisk/Genevant, AbbVie/Aldeyra and more.

Great Britain is first to approve the groundbreaking CRISPR-based therapy for sickle cell and beta thalassemia. Vertex is yet to reveal its price, however, and wants to play down early expectations of its commercial potential.