The US Food and Drug Administration (FDA) has expanded the use of Casgevy (exagamglogene autotemcel) for the treatment of people ages two years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). The approval makes Casgevy the first… Read More
Vertex Pharmaceuticals has announced data demonstrating the clinical benefits of Casgevy (exagamglogene autotemcel) in people ages five years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, from pivotal studies in children ages 5–11, show that the efficacy and safety… Read More
Biotech Cellarity has dosed its first patient in a Phase I clinical trial of CLY-124, a first-in-class Globin-Switching oral medicine designed to treat sickle cell disease (SCD). “Initiation of our first-in-human clinical study for CLY-124 represents the delivery of our first clinical asset from our platform with… Read More
The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib for sickle cell disease (SCD). Rilzabrutinib is an oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor that works via multi-immune modulation, to target a reduction in vaso-occlusive crises, which may occur… Read More
Biotech Tessera Therapeutics has presented updates across its in vivo genetic medicine programmes for Alpha-1 antitrypsin deficiency (AATD), Phenylketonuria (PKU), and Sickle Cell Disease (SCD), as well as advances in in vivo T cell therapies. These data were shared across four oral presentations and three poster presentations at the American Society of Gene and… Read More
Tessera Therapeutics, a biotech developing a new approach to genome engineering, has entered into an agreement with the Bill & Melinda Gates Foundation to jointly fund the company’s in vivo programme for sickle cell disease (SCD). The funding includes an initial investment, with potential total investment of up to $50… Read More
The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology. Read More
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… Read More
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… Read More
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… Read More
