Gene therapy has changed the drug development process, opening new doors to commercial opportunities for sponsors through innovative tools, processes and drugs. The advent of practical gene editing has allowed us to center on a patient's genetics to develop specific gene-targeted medicines. In the early stages of clinical development, gene editing tools have been indispensable to advancing patient-centric cell and gene therapies to date, enabling to rapidly grow and select the right toolset to address a specific disease is critical for success. Yet, these tools are only a means to an end, and potentially greater challenges remain or will arise. Even though this form of medicine currently enjoys the great public promise of cutting-edge tools and improved clinical outcomes, finding, recruiting and following patients for these trials is proving increasingly difficult due to a variety of reasons. To effectively address patient-centric genetic medicine risks and realties, the following challenges must be answered in your clinical development plan: What is the right type of therapeutic for a particular disease? What are the strengths and/or limitations of your technology? How will you profile and then find the right patients for your trial in terms of patient burden relative to disease? Once found, what data-driven, patient-centric approaches will you use to effectively and efficiently enroll cohorts in your study? In this webinar, we will attempt to provide a specific framework of information and suggestions to address these questions.
Join our upcoming webinar to learn more about:
- Personalized gene therapy: key concepts and disease areas that will benefit
- Finding and recruiting the right patients for your cell and gene therapy assets
- Promises and challenges in treating and following patients treated with genetic medicines
- Understanding the opportunities and risks of your technology in the gene editing trial setting and beyond