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.
Whether the experts come from a CRO or a regulatory consultancy, their help will be critical in helping develop the trial, meet with the Institutional Biosafety Committee, establish a timeline, and manage expectations. Here’s why.
Even measured against the vast scientific mystery that defines the biotech industry, gene therapy poses extraordinary challenges. This paper explores the history of gene therapy trials, as well as the types of gene therapy vectors and delivery strategies. Also discussed is the regulatory and operational challenges associated with gene therapy trials, including start-up regulations, site selection, recruitment, and retention.
Both registry studies and natural history studies play important roles in rare disease research. Understanding the differences between the two types of studies and how they can be used to inform clinical development can help sponsors plan for success.
A trans-Atlantic study to evaluate an antibody for treatment of B-cell non-Hodgkins lymphoma overcame patient recruiting challenges and has already succeeded beyond expectations in the form of five patients declared disease-free a year and counting while still three years from completing patient follow-up.
Many biopharmaceutical companies have made significant investments in research and the clinical development of gene therapies. For those working to develop and bring new gene therapy treatments to market, it is critical to select the right contract research organization (CRO) to conduct these studies. Here are three helpful strategies to follow when selecting a CRO partner.
Delivering cell and gene therapies is an expensive and highly complex process, and there are a number of critical metrics that manufacturers should consider when selecting treatment sites. Subsequent activities — preparing sites to receive and initiate therapy, and managing site training and ongoing certification — can be equally, if not more, challenging.
Mary Elizabeth Williams is a journalist and an author. She is also a patient who has with metastatic melanoma who participated in a Phase 1 immunotherapy clinical trial. There is much that pharma can learn from her in terms of how companies approach clinical trials and drug development.
Process characterization is an important step in the overall journey of product development. In this poster, scientists at propose a step-wise approach for the definition and execution of process characterization for the production of a monoclonal antibody.
Gene therapy may be the next revolution in healthcare. The opportunity to cure diseases through the modification of the genome was once considered science fiction, but no longer. The United States has seen a few approvals in this area, including the approval of voretigene neparvovec for a rare form of blindness. In addition, CAR-T agents, two of which are available, have demonstrated alternative ways to treat disease by manipulating genes of different cells.