CGTLive’s Weekly Rewind – June 17, 2022

Welcome to CGTLive’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

1. T-Charge CAR T-Cell Therapy Shows Efficacy in Multiple Myeloma

Novartis’ BCMA-targeted chimeric antigen receptor (CAR) T-cell therapy PHE885 has shown efficacy in relapsed/refractory multiple myeloma (R/R MM).

2. Frederick Locke, MD, on Further CAR T Research

The vice-chair of the Department of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center discussed more work to be done with CAR T-cell therapies.

3. Clinical Hold Lifted on Homology’s pheNIX Trial for Phenylketonuria

The FDA has lifted the clinical hold on Homology Medicines’ phase 1/2 pheNIX clinical trial (NCT03952156) of the gene therapy HMI-102 for the treatment of phenylketonuria (PKU).

4. Massimo Trucco, MD, on Improving Access to Islet Cell Transplant

The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed iselt cell transplant for the treatment of chronic pancreatitis.

5. Vutrisiran Approved for hATTR Amyloidosis

The FDA has approved Alnylam’s investigational subcutaneous RNA interface therapy vutrisiran, marketed as Amvuttra, for the treatment of transthyretin-mediated (ATTR) amyloidosis.