In September 2024, CGTLive tracked promising data updates in CAR-T therapy for DLBCL, gene therapy for XLRP, and more. REGENXBIO's RGX-121 showed efficacy in MPSII, Biogen's nusinersen demonstrated efficacy in SMA at a higher dose, Allogeneic CAR-T azer-cel achieved CRs in DLBCL, Beacon's AGTC-501 maintained a favorable benefit-risk profile in XLRP, and Adicet Bio's ADI-001 indicated potential for autoimmune disease treatment.