Tag: gene therapy

Medical and Regulatory Affairs

The Guardrails of GMOs: Regulatory Considerations for Gene Therapy Trials

Like any new medical product, gene therapies must undergo rigorous approval processes. However, there are a number of additional regulations for gene therapies that don’t apply to conventional pharmaceuticals. This is especially true within the European Union. (It’s important to note that while most gene therapy studies are based in the U.S. and Europe — which...

Clinical Research: Phase 1 - Phase 4

Gene Therapy 101: From the 1960s to Today

Gene therapy is a hot topic in clinical research today — and for good reason! These technologies have the potential to treat — and in some cases even cure — a wide range of conditions, including rare genetic disorders that previously had no effective therapies. What Is Gene Therapy? Gene therapies are a diverse group of...

Medical and Regulatory Affairs

The Regulatory Landscape for Immuno-gene Therapeutics in Hematological Malignances

Over the past few years, there has been a dramatic increase in the use of genetically modified cells for cancer immunotherapy, including chimeric antigen receptor (CAR)-T cells, recombinant T-cell receptor T cells and genetically modified CD34+ cells for the treatment of hematological malignancies. Clinical trials of immuno-gene therapeutics are becoming increasingly common, and regulatory guidelines...