Category: Gene therapy

According to clinical trial data, valoctocogene roxaparvovec (brand name Roctavian) demonstrated a significant reduction in annualized bleeding rates for men with severe hemophilia A. This innovative treatment represents a major advancement in the management of this genetic bleeding disorder by addressing its root cause through gene therapy.

Metachromatic leukodystrophy (MLD) is a rare inherited disease, and for eligible patients, the gene therapy Libmeldy offers a potentially curative, one-time treatment. The process to administer this revolutionary therapy is complex, involving several meticulous steps from initial cell collection to final intravenous infusion at a qualified treatment center.

Affecting approximately one in every 100,000 live births, metachromatic leukodystrophy (MLD) is a devastating genetic disorder. For eligible patients, the innovative one-time gene therapy, Libmeldy, offers a new approach to treatment, but understanding **how does libmeldy work** requires a deep dive into advanced molecular science.

Gendicine was the first commercial gene therapy product for cancer approved globally by the Chinese State Food and Drug Administration in 2003. The therapy, often known as recombinant adenovirus p53 or rAd-p53, targets malignant cells by delivering a functional copy of the critical p53 tumor-suppressor gene.

In a landmark decision in late 2023, the U.S. FDA approved Casgevy, the first treatment utilizing CRISPR technology to receive authorization. Understanding **how does casgevy gene therapy work** involves a look at its process of gene editing to enable a patient's body to produce healthy blood cells.