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
Drug Discovery World speaks to key players in the cell and gene therapy sector to learn about the opportunities and challenges in this promising but difficult treatment area. … Read More
The European Commission (EC) has granted approval for Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) for the treatment of people with cystic fibrosis (CF) ages six years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “Thousands of people with CF across… Read More
Vertex Pharmaceuticals has shared new Phase I/II data for zimislecel (VX-880), an investigational stem cell-derived, fully differentiated islet cell therapy. The FORWARD-101 clinical trial is investigating the treatment in people with type 1 diabetes (T1D) with impaired hypoglycaemic awareness and severe hypoglycaemic events (SHEs). All… Read More
Vertex Pharmaceuticals has shared data demonstrating the positive clinical and quality of life benefits of treatment with cystic fibrosis transmembrane conductance regulator (CFTR) modulators and in particular, Alyftrek (deutivacaftor/tezacaftor/vanzacaftor). These data were presented at the European Cystic Fibrosis Society’s (ECFS) 48th European Cystic Fibrosis Conference held 4-7… Read More
Vertex Pharmaceuticals and Orna Therapeutics have announced plans to partner on gene therapies for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The three-year strategic research collaboration will utilise Orna’s lipid nanoparticle (LNP) delivery solutions. In return Orna will receive upfront payments of $65… Read More
A cystic fibrosis drug targeting the basic defect that causes the condition has been shown to be safe and effective in newborns aged four weeks and above, new research has found. The study, involving RCSI University of Medicine and Health Sciences and Children’s Health Ireland, included… Read More
The top 20 global biopharmaceutical companies have reported 1.6% market capitalisation growth to $3.67 trillion in 2023, according to data analysts GlobalData. The top 20 global biopharmaceutical companies experienced varied year-on-year (YoY) market capitalisation shifts in 2023 amid the macroeconomic headwinds, steep patent cliffs and the… 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
A label expansion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) has been approved in the European Union (EU). The European Commission has extended use of the therapy to include children with CF aged two through five years… 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
