In recent years, immunotherapy has led to substantial advances in cancer therapy. In particular, the immune checkpoint inhibitors — PD-1/PD-L1 and CTLA-4 inhibitors — have revolutionized treatment for certain hematologic malignancies and solid tumors. The U.S. Food and Drug Administration...
Gene therapy holds great promise as a potential treatment for Parkinson’s disease, a disorder for which currently available medications do not causally treat the underlying disease mechanisms. However, given the early stage of gene therapy research in this therapeutic area,...
Gene therapy development is gaining momentum. In 2020 alone, gene therapy attracted $12 billion in global financing through just the first three quarters with more than 370 gene therapy clinical trials underway worldwide.1 The U.S. Food and Drug Administration (FDA) itself...
Background Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad of challenges of operationalizing these studies will help you bring safe, breakthrough treatments to patients with unmet medical needs. Premier Research...
Though gene therapy has been around for decades, it continues to pose extraordinary challenges in the areas of R&D, clinical development, and operation of clinical trials. Sponsors and CROs face shifting operational and regulatory demands amid rapid advances in the...
Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help you bring safe, breakthrough treatments to patients with unmet medical needs. In this blog post,...
As scientific knowledge, clinical experience, and acceptance of gene therapy products have evolved, so have the regulatory frameworks for ensuring the safety of these novel treatments. To date, there is no harmonized international standard for regulating gene therapy products; however,...
Gene therapy products are typically designed to achieve therapeutic effect through long-acting or permanent changes in the human body. Due to this extended period of exposure, patients who receive gene therapies may be at increased risk of delayed adverse events....
Skin contains essential stem cell populations and other cell types that are critical for renewing and maintaining its structural integrity and function.1 Of the many types of skin stem cells that have been identified, epidermal stem cells — primarily keratinocyte...
When it comes to dermatologic conditions, gene therapy is still in its very early stages. However, we are seeing promising potential solutions for some rare genetic dermatology diseases, as well as ongoing research in more common skin conditions. The success...