Cell and Gene Therapies (CGTs) are shaping the future of medicine offering groundbreaking treatments for patients battling with diseases that span from rare genetic disorders to oncological and long-term chronic conditions. The significant advancements made in the recent years yet need to face several unique challenges, such as addressing the risk presented by extractables and leachables (E&L).
CGTs are primarily manufactured via single-use systems (SUS) which consist of many different components, including bags, tubing, filters, pumps, etc. The compounds possibly released from these materials can have a direct and significant impact on both the safety and the potency of the CGT final products. When cells contact the SUS indeed, it can cause the release of extractable substances, referred to as process equipment-related leachables (PERLs) that may accumulate throughout the manufacturing process, potentially compromising the quality of the product and the patient safety.
Given the potential for these compounds to accumulate, a thorough E&L testing strategy should extend beyond the final stages of drug product manufacturing to encompass earlier manufacturing steps as well. Regulatory authorities require extractables and leachables testing of any materials in the manufacturing stream that come into contact with the drug product and the most effective way to ensure that SUS are suitable for the intended use, is to select well-characterized components. Of note, although not mandatory, where appropriate, considerations for E&L testing may also extend to critical raw materials: indeed, the complex biological structure of cells and viral vectors can have important, and difficult to predict, impacts on E&L accumulation and metabolization.
While USP chapters 665 and 1665 provide valuable guidance for extractable and leachable (E&L) testing in biological products, they lack specific recommendations for C> therapies. Therefore, a tailored strategy for risk assessment and analytical activities is necessary.This webinar will delve into the challenges faced by manufacturers in developing a successful E&L testing strategy, offering practical advice on study design and result interpretation. Our expert speakers will provide insights into handling E&L studies for components that are specifically utilized in cell and gene therapies, in order to meet regulatory requirements and expectations, and ultimately ensure product quality and patient safety. In particular the webinar will address the risk of extractables and leachables in cell therapy manufacturing and how the new USP<665> and <1665> chapters could be adapted to CGT products.
The webinar will establish the foundation for manufacturers of cell and gene therapies on what to expect when it comes to regulatory requirements and develop a study program that will assist them in achieving approval from regulatory authorities when they submit their packages.