Reflection paper on design modifications of gene therapy medicinal products during development

The committee for advanced therapies' of the European Medicines Agency reflection paper on management of clinical risks deriving from insertional mutagenesis.

In the European Union, the Committee for Advanced Therapies of the European Medicines Agency takes the lead in the scientific assessment for marketing authorization applications for advanced therapy medicinal products, which include gene therapy medicinal products, somatic cell therapy medicinal products, and tissue-engineered products. The Committee for Advanced Therapies also takes the lead i...

Reflection paper on the pharmaceutical development of intravenous medicinal products containing active substances solubilised in micellar systems

The Role of Gene Therapy in Cartilage Repair

The key principle of gene delivery to articulations by direct intra-articular injection is to release complementary DNA(cDNA)-encoding medical products that will lead to maintained, endogenous production of the gene products withinthe articulation. In fact, this has been accomplished for both in vivo and ex vivo gene delivery, using several vectors,genes, and cells in some animal models. Some c...

EMEA and Gene Therapy Medicinal Products Development in the European Union

The evaluation of quality, safety, and efficacy of medicinal products by the European Medicines Evaluation Agency (EMEA) via the centralized procedure is the only available regulatory procedure for obtaining marketing authorization for gene therapy (GT) medicinal products in the European Union. The responsibility for the authorization of clinical trials remains with the national competent autho...

Design and development of CAR-T cells for cancer therapy

Introduction: Today, treatment with CAR-T cells is accepted as an effective treatment for blood malignancies. CAR-T cells are autologous T cells that are engineered by gene transfer techniques to express a chimeric antigen receptor (CAR). Despite the promising results and the approval of six CAR-T cell products; these products have not yet been approved for solid tumors. In addition, the high c...