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It was a monumental week in the gene and cell therapy space that featured an FDA approval for a renal cell carcinoma therapy and two FDA designations, one for a treatment for patients with leukocyte adhesion deficiency-I and another for hemophilia A. With new treatments on the horizon for rare diseases, the evolution of CAR T-cell therapy in cancer care, DNA-based Immunotherapy and many more innovations, this year is shaping up to be a groundbreaking one for patient care. Be sure to stay tuned to GeneTherapyLive.com for up-to-the-minute breaking news in gene and cell therapy, interviews with industry leading KOLs and more.
 
 
CAR T Therapy Induces Response in Majority of Patients With Relapsed Multiple Myeloma
Idecabtagene vicleucel induced progression-free survival up to 20 months and a complete response in one-third of patients with multiple myeloma who had relapsed multiple times and had received at least 3 previous lines of treatment.
 

 
D’Angelo Describes Innovative Combos Designed to Produce Durable Activity in Relapsed/Refractory DLBCL
Christopher D'Angelo, MD, discusses emerging CAR T-cell therapies in the space, as well as in follicular lymphoma, and the challenges with using a 5-drug regimen.
 

 
FDA Grants RP-L201 RMAT Designation for LAD-I
The FDA has granted an RMAT designation to RP-L201 as a potential treatment for patients with leukocyte adhesion deficiency-I.
 

 
mRNA-Based CAR T-Cell Therapy Descartes-11 Moves Into Phase 2 Trial in Myeloma
A phase 2 clinical trial has been initiated to evaluate Descartes-11, an mRNA CAR T-cell therapy, as a consolidative therapy in patients with newly diagnosed, high-risk multiple myeloma who have residual disease after induction therapy.
 

 
FDA Grants Valoctocogene Roxaparvovec RMAT Designation for Hemophilia A
The FDA has granted an RMAT designation to valoctocogene roxaparvovec for hemophilia A.
 

 
AXO-AAV-GM2 Administered to First Patient in GM2 Gangliosidosis Trial
The 2-stage study is an open-label, 2-stage clinical trial designed to evaluate safety and dose-escalation (stage 1) and safety and efficacy (stage 2) of surgically delivered AXO-AAV-GM2.