Glybera is the first gene therapy drug recommended for approval in the European Union. Gene therapy drugs have that potential. Gene therapy drugs have the potential to cure genetic disorders by replacing a faulty gene with a functional copy, helping the body regain its functionality. Glybera uses an adeno-associated viral vector as a delivery vehicle to add functional copies of the LPL gene to muscle cells to allow for the production of the enzyme in the cells.
Despite these problems and difficulties, the final opinion in favor of Glybera is encouraging news for the gene and cell therapy communities, and it is to be hoped that the evaluation of the twists and turns of the plot of this saga will help to streamline the regulatory processes of other gene and cell therapy products so that this new area of medicine can eventually fulfill its promise in human medicine. The US FDA criticized the trial design, suspended the university's entire gene therapy program (one of the largest in the world at the time) and began investigations into 69 other gene therapy trials that were being carried out across the country. San Raffaele Telethon Institute for Gene Therapy (SR-TIGET): based in Milan (Italy), the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) is a joint venture between Ospedale San Raffaele and Telethon. Here you will find articles on molecular therapy, courtesy of the American Society for Gene and Cell Therapy.
Among these products, a chondrocyte cell therapy product was finally approved, while adenoviral thymidine kinase gene therapy was not approved for malignant glioma. The specialized Advanced Therapies Committee (CAT), created by the EMA to review gene and cell therapy products, advised against the marketing authorization of Glybera, so the Committee for Medicinal Products for Human Use (CHMP), which makes final recommendations for marketing authorization in the EU.
