CGTLive’s Weekly Rewind – January 13, 2023

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. B-VEC PDUFA Date Extended to May 2023

The FDA made the decision in response to manufacturing process information that it considered a major amendment.

2.Rebecca Epperly, MD, on Emerging Clinical Research With Cell Therapy in Leukemia, Lymphoma

The clinician scientist at St. Jude Children’s Research Hospital discussed the session on cell therapies in acute leukemias and Hodgkin lymphoma she moderated at ASH 2022.

3. Sickle Cell Disease Gene-Editing Therapy Trial Voluntarily Paused Following Serious Adverse Event

The first patient dosed in the study experienced a case of prolonged pancytopenia that has required ongoing transfusion and growth factor support.

4. Giedre Krenciute, PhD, on Revitalizing T-cells in CAR T-cell Therapy

The assistant member of the bone marrow transplantation and cellular therapy department at St. Jude Children’s Hospital discussed preclinical research presented at ASH 2022.

5. Val-rox Meets End Points in Hemophilia A at 3 Years

The new analysis data was requested by the FDA ahead of the therapy’s March 31, 2023 PDUFA date.