Sickle cell disorder (SCD)

CRISPR gene editing

Great Britain first to approve CRISPR-based gene therapy

16 November 2023

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). Casgevy (exagamglogene autotemcel, or exa-cel) is the first medicine to be licensed that uses gene editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. The treatment from […]

This week in drug discovery (11-15 July)

15 July 2022

News round-up by DDW’s Reece Armstrong for 11 – 15 July. Urgency a must for global fight against AMR, report says Urgent access to vaccines that can fight and prevent infections, and curb the risk of antimicrobial resistance (AMR) is needed to save lives, according to a report by the World Health Organisation (WHO). Could […]

First treatment for sickle cell side-effect in 20 years approved

13 July 2022

The first new treatment in 20 years that prevents a common, painful side-effect in patients with sickle cell disorder (SCD) has been issued interim acceptance by the Scottish Medicines Consortium (SMC).  The SMC issued interim acceptance for Novartis UK’s Adakveo for use in NHS Scotland as a treatment option for the prevention of recurrent vaso-occlusive […]