CGTLive’s Weekly Rewind – November 17, 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. This week’s coverage includes some highlights from the recent American Heart Association meeting.

1. MHRA Approves Gene-Edited Cell Therapy Exa-cel for Sickle Cell and Transfusion-Dependent β-Thalassemia

Vertex and CRISPR’s Exa-cel, which remains an investigational treatment in the United States, is the first CRISPR-based gene therapy to be approved in the UK, where it will be marketed as Casgevy.

2. Ben Creelan, MD, on the Potential of Logic-gated Tmod CAR-T in Lung Cancer and Other Solid Tumors

The medical oncologist at Moffitt Cancer Center spoke about A2 Bio’s A2B530 as part of a larger discussion of novel cell therapy approaches in solid tumors.

3. Sanfilippo Awareness Day: A Peek Into the CGT Pipeline

CGTLive takes a look at MPSIII therapies in the pipeline for World Sanfilippo Awareness Day on November 16.

4. Xaviar Michael Jones, MD, on the Potential to Address Unmet Needs in Systemic Sclerosis With RNA Therapeutics

The postdoctoral research fellow at Cedars Sinai Medical Center discussed preclinical research he presented at the American Heart Association’s Scientific Sessions 2023.

5. Single RNAi Therapy Injection Controls Mild-to-Moderate Hypertension for 6 Months

Data on zilebesiran from the KARDIA-2 trial, in combination with other hypertension medicines, are expected in 2024.