Gene therapy technologies (including gene editing) and genetically modified organisms face special operational and regulatory challenges for the conduct of clinical trials. This is particularly true for global trials that face a myriad of national approvals in addition to the regulatory agency and ethics committee approval. Recognizing the potential of gene therapies, regulators have implemented frameworks that support the development and approval these therapies for serious disease. With an increased emphasis on conducting global clinical trials the investigation of the diverse additional requirements becomes paramount for developing a time- and cost-effective regulatory strategy. Additionally, gene editing technology carries a concern of unethical use of the technology as was exemplified by the birth of the germline edited twins, Nana and Lulu in 2018. Germline editing has been prohibited by many jurisdictions and is often included in new frameworks that are being established for advanced therapies. The session will discuss case studies on various gene editing technologies including ongoing clinical trials and provide overview of sample national frameworks to support overcoming operational and regulatory development challenges. Session speakers offer their insights and experiences in tackling challenges at various stages of the development program from perspectives of various stakeholders (e.g. industry, regulators). What are the technologies involved and regulatory requirements how are they applied? What are the ethical considerations? What opportunities for interactions with the regulators were taken?
Three industry experts will share their experience in developing therapies involving gene editing technologies addressing pitfalls, hurdles, successes and lessons learned. The audience will have ample time to ask questions and support the session by providing responses to polls throughout the session, discussing their experience during the final panel discussion/Q&A.
Describe the regulatory, development and ethical challenges associated with gene editing technologies.
Explain the regulatory considerations for global clinical trials and ethical considerations/restrictions in various jurisdictions.
Identify regulatory support options to optimize development plans for time and cost savings.