One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). PDUFA VII, as the reauthorization is commonly known, will cover the fiscal years 2023-2027. Many of...
The gene therapy era can be said to have begun in 1990, when the first gene therapy clinical trial took place. Some 3,000 clinical trials have followed that first study, a resounding affirmation of innovators’ increasing recognition of gene therapy’s...
Immunotherapy has led to substantial advances in cancer therapy in recent years. Still, unpredictable response rates and immune-related adverse events have hampered the widespread use of immune checkpoint therapy to treat cancers. To tackle these challenges, sponsors are increasingly looking...
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,...