Cell & Gene Therapy

Approved CRISPR therapies and a maturing gene-therapy pipeline reshape genetic disease treatment with multiple FDA approvals in the past two years. The session covers Casgevy (exa-cel) and Lyfgenia in sickle cell disease, allogeneic CAR-T platforms (Caribou, Allogene, Precision BioSciences), AAV capsid engineering for organ-targeted delivery, in vivo CRISPR base editing (VERVE-101 and VERVE-102), and the manufacturing and reimbursement frontiers including outcome-based contracts. Discussion addresses cell-therapy CMC scaling, decentralised manufacturing models, the regulatory pathway for in vivo gene editing, and the ethics of germline editing as the technology matures.