Gene Therapy for Hematological Disorders

Gene therapy, a molecular medicine based on vector-mediated transfer of therapeutic genes, holds promise for a cure of monogenetic inherited diseases. In recent years, tremendous progress has been reported in the treatment of hematological disorders. Assessment of the safety of gene therapy medicinal products must be based on scientific findings and critical unbiased judgement of experimental data or evidence, and should not be influenced by political expediency, legal advice or regulatory sensitivities/prejudices. Safety testing must be specific for individual vector types and all tests must be well-validated using suitable reference materials. All tests that are carried out must be relevant to the actual and perceived safety risks of vectors. Unnecessary tests for non-existent risks should be avoided. Most safety testing that is currently applied to gene therapy medicinal products is carried out by manufacturers and academic or pharmaceutical developers. Independent testing laboratories capable of performing platform safety studies addressing common important risks are few and far between. More funding should therefore be allocated to support independent testing of gene therapy medicinal products to ensure safe use of gene therapy medicines and to support future developments.