Cryopreservation and Logistics of Advanced Therapy Medicinal Products: A Cold Subject Suddenly Becomes a Hot Topic

Advanced therapy medicinal products (ATMPs) are ‘living drugs’ that have proven highly effective at treating diseases, such as cancer, that were once thought untreatable. As ‘living drugs’, ATMPs require complex chemistry, manufacturing, and controls (CMC) to enable efficient commercialization and maintain therapeutic consistency and potency. Part of these controls is the need to implement optimizing freezing protocols to facilitate the long-term frozen storage and transport of ATMPs from the manufacturing site to the clinic. Cryopreservation for ATMPs requires specialized storage conditions and distribution channels to maintain ATMPs at ultralow temperatures (e.g. below -80°C) that permit proper biological function post-thaw. As evidenced by the recent COVID vaccine approvals, cryogenic storage and distribution at ultralow temperatures is a challenge for the existing pharmaceutical infrastructure and a hinderance to the widespread distribution of ATMPs. Solutions to overcome the challenges of cell cryopreservation, storage, and distribution at ultralow temperatures need to be identified, validated, and implemented to increase ATMP adoption for the general population. This presentation will introduce the audience to the challenges associated with ATMP cryopreservation, as well as provide potential solutions for how these challenges can be tackled during ATMP development, from pre-clinical through commercialization.