Review top news and interview highlights from the week ending 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.
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.
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.
CGTLive takes a look at MPSIII therapies in the pipeline for World Sanfilippo Awareness Day on November 16.
The postdoctoral research fellow at Cedars Sinai Medical Center discussed preclinical research he presented at the American Heart Association’s Scientific Sessions 2023.
Data on zilebesiran from the KARDIA-2 trial, in combination with other hypertension medicines, are expected in 2024.
Will the CMS CGT Model Support Patient Access to Sickle Cell Disease CGTs?
July 25th 2024Cell and gene therapies can transform the paradigm of care for patients with chronic, complex conditions, but these therapies come at an up-front cost of several million dollars per treatment, complicating the pipeline of access to them.