CGTLive’s Weekly Rewind – December 16, 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. Beti-Cel Confers Durable Efficacy, Improved Quality of Life in Beta-Thalassemia

100% of patients who achieved transfusion independence reported an overall benefit from treatment.

2. David Sallman, MD, on Advantages of Allogeneic CAR T-Cell Therapy in AML

The assistant member of the department of malignant hematology at Moffitt Cancer Center discussed unmet needs in acute myeloid leukemia and how UCART123v1.2 may address these.

3. GPRC5D-Targeted CAR T-Cell Therapy Shows Promising Efficacy After BCMA Therapy in R/R MM

New data from a phase 1/2 trial of BMS-986393 were presented at the 64th Annual ASH Meeting.

4. Akshay Sharma, MBBS, on Inducing Fetal Hemoglobin in Sickle Cell With CRISPR/Cas9-Edited Stem Cells

The assistant member of the bone marrow transplant department at St. Jude Children’s Research Hospital discussed new data from a phase 1/2 trial presented at ASH 2022.

5. SPK-8011 Yields Durable FVIII Expression in Hemophilia A

Annualized bleed rate and FVIII infusion rates significantly decreased after infusion with the gene therapy.