Gene Therapy Medicinal Products

Gene therapy medicinal products are innovative treatments that aim to treat or prevent diseases by modifying the genetic material within a patient’s cells. Typically, these therapies work by adding, editing, or silencing genes that cause or contribute to diseases.

1. Gene Addition: A healthy gene is introduced into a patient’s cells to replace a faulty or missing gene, used in genetic disorders like cystic fibrosis or inherited blindness.
2. Gene Editing: Precise alterations are made to a patient’s existing genetic material to correct mutations directly, using technologies like CRISPR-Cas9. This method holds promise for curing genetic diseases at the DNA level.
3. Gene Silencing: This involves turning off or suppressing a gene that is causing harm, such as in some cancers where a gene produces harmful proteins.

Gene therapy products are often delivered using modified viruses that carry the therapeutic gene into cells, though non-viral methods are also being explored. These therapies are applied to a range of conditions, including inherited genetic disorders, certain cancers (e.g., blood or lymphatic cancers where CAR-T cell therapies are applied), and also infectious diseases like HIV.