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. Developers of cell and gene therapies (CGTs) have had a difficult few years. Despite the undoubted promise of exciting new technologies in this area to… Read More
CRISPR Therapeutics has shared positive Phase I data from an ongoing clinical trial evaluating CTX310, an investigational, in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3. A single-course treatment with CTX310 produced dose-dependent, durable reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum… 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. However, the FDA added a black… Read More
Scientists have used CRISPR gene editing to target and rapidly destroy glioblastoma cells in an approach that could apply to other highly mutated cancers. Using a technique they’ve dubbed “cancer shredding,” the researchers at Gladstone Institutes programmed CRISPR to zero-in on repeating DNA sequences present only… 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 awarded the Nobel Prize… Read More
