There are many special circumstances and considerations when planning a gene therapy trial. Whether the experts come from a CRO or a regulatory consultancy, their help will be critical in developing the trial, meeting with the Institutional Biosafety Committee, establishing a timeline, and managing expectations. Here’s why.
Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. With the rapid evolution of the gene therapy field, regulatory agencies have been working to keep pace with these scientific and clinical breakthroughs. An understanding of the regulations and guidance documents reflecting regulator current thinking surrounding gene therapies is essential to success.
Researchers have made many attempts at disease modification as they pursue breakthroughs in treating Parkinson’s disease, but so far without success. Why have these efforts failed, and what’s next in treating this degenerative disorder that affects an estimated 10 million people worldwide? This article examines these past attempts, why they failed, and what targets are currently being pursued.
Sponsors of emerging hematologic therapies — including gene transfer and gene editing, adoptive cellular, and antibody-drug conjugates — face a unique set of challenges in conducting early-phase, dose-finding studies. Key considerations for developing early-phase trials can more accurately define the recommended dose and identify adverse events for emerging therapies.
Even measured against the vast scientific mystery that defines the biotech industry, gene therapy poses extraordinary challenges. This article explores the history of gene therapy trials, as well as the types of gene therapy vectors and delivery strategies. Also discussed are the regulatory and operational challenges associated with gene therapy trials, including start-up regulations, site selection, recruitment, and retention.