Good News! 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.
US Food and Drug Administration (FDA) 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.
In high-income economies, gene therapy is much easier to adopt. However, the situation is different in lower-income economies. Only few pharmaceutical companies are making an effort in these countries, there is virtually no legislation specific to gene therapy, while the medical experts are facing challenges in local clinical trials. Other obstacles to gene therapy in Africa include the following: it is a new approach and the danger of implementation could be high at this level, some ethical issues and policies are yet to be implemented, the technological know-how and know-well needs to be addressed, lack of expertise and low medical literacy. In addition, inadequate infrastructure leading to delayed diagnosis and treatment and cost-effectiveness of the project, standard of medical care, herbal medication, and spiritual healing ideologies represent a relevant issue.
Gene therapy brings solutions to treatments for life-threatening. Nonetheless, the sponsors of these products encounter difficulties in proving their safety and efficacy since they take non-traditional applications and implications.
