The European Medicines  Agency (EMA) has recommended conditional approval of Casgevy, the first medicine using CRISPR/Cas9, a novel gene-editing technology.  It is indicated for the treatment of severe Sickle Cell Disease (SCD) and transfusion-dependent beta-thalassemia in patients 12 years of age and older for whom haematopoietic stem cell transplantation is appropriate and a suitable donor is not available.

Casgevy was supported through EMA PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients' unmet medical needs.

US Food and Drug Administration also approved Casgevy and Lyfgenia for the treatment of SCD patients 12 years of age and older with recurrent vaso-occlusive crises.

Casgevy is a cell-based gene therapy medicinal product using CRISPR/Cas9 technology to edit the patient's own hematopoietic stem cells leading to improvements of patients’ quality of life.

Lyfgenia uses a lentiviral vector (gene delivery vehicle) for genetic modification.

Gene therapy involves introducing, removing or editing one or more genes to treat, prevent, or manage diseases, with the primary objective of restoring or enhancing normal cellular and tissue function. Particularly, gene editing could enable faulty genes to be corrected without providing a healthy copy externally, and simultaneous action on the genome at several points also working on conditions caused by mutations in several genes.

These advanced technologies are very promising for the treatment of genetic disorders including also rare diseases; however, several concerns should be considered such as their short- and long-term safety and efficacy, immune responses and genome manipulation, complexity of manufacturing, costs as well as patients’ accessibility.

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