This week in drug discovery (13-17 November) 

News round-up for 13-17 November by DDW Digital Content Editor Diana Spencer.

The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing. However, there were a number of other breakthroughs announced in the cell and gene therapy field.

The top stories:

Single-course in vivo base editing therapy proven to lower cholesterol

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101.

CAR-T therapy Yescarta reduces risk of death by more than 27%

New trial evidence has shown that Kite’s CAR-T cell therapy Yescarta (axicabtagene ciloleucel: axi-cel) reduces the risk of death by 27.4% in patients with relapsed or refractory large B-cell lymphoma (R/R LBCL), corresponding to a 38% improvement in overall survival (OS).

Great Britain first to approve CRISPR-based gene therapy

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT).

One-time gene therapy could change treatment of heart failure

Asklepios BioPharmaceutical (AskBio), a gene therapy subsidiary of Bayer, has presented first-in-human data from a Phase I trial investigating AB-1002 (also known as NAN-101) for the treatment of patients with congestive heart failure (CHF).

CART-ddBCMA Phase I trial shows durable response in myeloma patients

Arcellx has revealed encouraging interim data from its Phase I study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma.

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