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 is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.
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