The UK’s Medicines and Healthcare Regulatory Agency (MHRA) has granted approval for Casgevy, the world’s first gene therapy for sickle cell disease, utilizing the CRISPR gene-editing tool. This landmark decision could bring relief to thousands of individuals in the UK suffering from this debilitating condition. Casgevy, developed by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics, is authorized for patients aged 12 and older with sickle cell disease and thalassemia. Until now, bone marrow transplants were the primary treatment, associated with severe side effects.
Casgevy targets problematic genes in a patient’s bone marrow stem cells, enabling the production of healthy hemoglobin. The therapy involves chemotherapy, stem cell extraction, genetic editing, and reinfusion, requiring at least two hospitalizations. MHRA’s approval is based on studies that showed significant pain reduction in sickle cell patients and a decreased need for red blood cell transfusions in thalassemia patients.
Gene therapy treatments can be expensive, but efforts are underway to negotiate access and reimbursement. The US FDA is currently reviewing Casgevy, with a decision expected soon. The therapy has the potential to benefit millions of people worldwide with sickle cell disease, particularly those from regions where malaria is prevalent.
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
[2024/12/20] Gene and Cell Therapy- weekly digest from PackGene
FeaturedNewsArticlesPackGene's NewsletterReceive the latest news and insights to your inbox.About PackGenePackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span...
Sangamo and Astellas Collaborate to Advance Neurological Gene Therapies Using AAV Capsid Technology
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a leader in genomic medicine, and Astellas Pharma Inc. (TSE: 4503), a global innovator in life sciences, have partnered under a new license agreement. This collaboration centers around Sangamo’s cutting-edge neurotropic AAV...
Inceptor Bio and GRIT Bio Announce Strategic Partnership to Advance IB-T101, a Next-Generation Solid Tumor CAR-T Utilizing the OUTLAST™ Platform
SHANGHAI and MORRISVILLE, N.C., Dec. 18, 2024 /PRNewswire/ -- Inceptor Bio, a leading innovator in cell therapy, and GRIT Bio, a clinical-stage immunotherapy developer, today announced a strategic partnership to advance IB-T101, a potentially best-in-class CAR-T...
Proof-of-concept study bioengineers therapeutics for improved cancer treatment
Credit: Pixabay/CC0 Public DomainA team of Children's Medical Research Institute (CMRI) scientists has identified a new method for producing a therapeutic product that has the potential to improve the treatment of cancer. The work by Associate Professor Leszek...
Related Services
