CGTLive’s Weekly Rewind – March 3, 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. Gene-Editing Therapy for Hereditary Angioedema Cleared for US Phase 2 Sites

Intellia is also seeking an IND for phase 2 US sites of its other lead candidate, NTLA-2001 for ATTR amyloidosis.

2. Amer Beitinjaneh, MD, MSc, MPH, FACP, on Treating EBV+ PTLD With Tab-cel

The associate professor of medicine at Sylvester Comprehensive Cancer Center discussed updated data from the phase 3 ALLELE study.

3. Hematopoietic Cell Transplantation Leads to Improved Cardiac Function in MPSI

Four of 5 patients showed improvements in ejection fraction following bone marrow transplant.

4. Marco Davila, MD, PhD, on Investigating Mechanisms of CAR T Resistance in B-cell Malignancies

The vice chair for cellular therapies at Roswell Park Comprehensive Cancer Center discussed new research from the center presented at the 2023 Tandem Meetings.

5. Upcoming FDA Decisions for Rare Diseases in 2023

CGTLive takes a look at some upcoming FDA decisions for Rare Disease Day.