Another exciting result is the interim result of the CARTITUDE-1 trial, a phase 1b/2 study of JNJ-4528, a CAR-T cell therapy containing two BCMA-targeting single-domain antibodies in relapsed and/or refractory MM, which demonstrated that at a target dose of 0.75 × 106 CAR+ cells/kg, it could deliver ORR of 91% with early and deep response, with a manageable safety profile [154]. The gene discussed is TNFRSF17; the disease is Miyoshi myopathy.