Gene Therapy
Gene therapy clinical trials are accompanied by their own sets of considerations and challenges. This is especially true when it comes to rare diseases, as there are fewer patients to enroll in clinical trials. Much like cell therapies, gene therapy products need to be stored and shipped properly using an appropriate chain of custody which requires unique tracking systems, equipment and monitoring, as well as logistical safeguards to ensure that the product reaches the site in the appropriate condition.
Gene Therapy Study Team Expertise
Caidya’s gene therapy trial management capabilities are overseen by a team of experienced professionals. The CGT Working Group oversees the execution of gene therapy trials and provides consulting services to the study team. Further, the CGT Working Group has created a suite of standardized tools, processes, plans, and technology which are leveraged by the study team in the planning, start-up and execution stages of the trial. Use of existing, standardized tools increases speed and efficiency allowing more time to focus on sites and patient management.
Experience Summary
Logistics considerations
Manufacturing and delivering gene therapies to study patients presents a unique set of challenges due to their temperature-sensitivity, personalized nature, and circular supply chain. To ensure safe delivery of these products to patients, sponsors must consider four areas: packaging engineering, hazardous material qualification, cold chain management, and chain of identity/traceability.
Caidya’s team of experts can help you develop a clear front-end planning strategy and well-defined sequence of activities and business processes related to packaging and temperature-controlled transport necessary to ensure safe and timely delivery of study product to sites for patient treatment during the clinical trial.
Regulatory Strategy
Gene therapy innovators face a number of challenging regulatory considerations when bringing treatments to market. Sponsors should take advantage of various programs from health authorities, such as PRIME from the EMA and FDA’s Breakthrough and Orphan Therapy Designations that can accelerate the development process for rare products with unmet medical needs. Early scientific advice before filing an application is essential and sponsors should reach out to the agencies early on to make the most of their interactions. Caidya provides insight and intelligence to Sponsors so they can evaluate and strategize an effective country approach that considers health authority expertise and Caidya’s lessons learned from previous services for similar products.
Our regulatory team has broad experience with global regulatory agencies in many countries and regions around the world. We have performed several gene therapy studies in the Americas, Europe and Asia and can provide expert guidance to stay ahead of new regulatory guidance and be proactive in providing clear information to authorities to avoid potential barriers.
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