GeneTherapyLive’s Weekly Rewind – December 17, 2021

Welcome to GeneTherapyLive’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. Beti-Cel Induces Transfusion Independence in Patients With β-thalassemia

The findings, which were simultaneously reported in the New England Journal of Medicine, support beti-cel as a potentially curative, one-time treatment option for these patients.

2. Anti-Thrombin siRNA Therapeutic Reduces Bleeding in Hemophilia

Over half of patients in the fitusiran arm of the ATLAS-INH study had 0 treated bleeding events.

3. Reprogramming the Immune System for Kidney Transplant and Scleroderma

Scott Requadt, chief executive officer, Talaris Therapeutics, discussed the company’s goal to reprogram the immune system. He discussed the company’s approach and how the COVID-19 pandemic has exacerbated the unmet need in these indications.

4. Assessing Axi-Cel in R/R LBC Lymphoma in ZUMA-7: Frederick Locke, MD

The vice chair, Blood and Marrow Transplant and Cellular Immunotherapy Program, and co-leader, Immuno-Oncology, Moffitt Cancer Center, discussed the results of the phase 3 ZUMA-7 trial.

5. Safety Advantages of Tab-Cel Over Other T-Cell Therapies in EBV+ PTLD

AJ Joshi, MD, chief medical officer, Atara Biotherapeutics, discussed safety findings from the phase 3 ALLELE study.