The International Society for Cellular Therapy (ISCT) is a global society of clinicians, regulators, technologists, and industry partners with a shared mission to drive the translation of all cell and gene (CGT) therapies for the benefit of patients worldwide. The ISCT 2023 Annual Meeting took place in Paris, France from May 31 to June 3 2023. DDW’s Megan Thomas heard from attendees about key trends in the sector. First, the importance of cell collections. Next, attendees discuss the trend of allo vs auto. Then, new generation MSCs. Here, attendees weigh in on future regulatory changes. The next article will cover redefining manufacturing.
According to Carole Nicco, Chief Scientific Officer of BioSenic, CGT products, regulated in the EU as Advanced Therapy Medicinal Products (ATMP), are among the fastest growing areas of scientific innovation and business in the life sciences today. She said: “Certainly, the novelty and complexity of this product modality requires a risk-based regulatory approach to define a de-risking plan. This regulatory strategy should be based on solid scientific data, taking into account general regulatory recommendations, to allow for a risk-benefit analysis adapted to the specificities of each CGT product. However, we need to avoid unnecessary delays in clinical development, and interactions between EU countries and with US regulators need to be homogenised if we want to interact with EU and US regulators.”
BioSenic’s had 44 centres open in Europe for the latest Phase IIb clinical trial for difficult tibia fractures, which is just finishing. Nicco said that even between the EU countries, the regulatory issues are difficult. She said: “For each change, the rules can vary from one EU country to another and everything has to be translated into the local language. That doesn’t help in recruiting patients and getting clinical results. The road from the validation of a drug to its use as a treatment is long and fraught with difficulties. Regulation is necessary, but optimising timescales would be ideal. Regulatory pathways, such as the Regenerative Medicine Advanced Therapy (RMAT) in the US and the Priority Medicines scheme (PRIME) in the EU, have been created to significantly accelerate the development process and review timelines for submissions. These new regulatory pathways include, among other benefits, more frequent interactions between developers and assessors/regulators to facilitate the exchange of new scientific and technological advances. Increased communication between developers and regulators has proven invaluable in areas where knowledge is still limited.”
Angela Osborne, CEO of eXmoor Pharma, has seen CMC requirements tighten and harmonise, for example in AAV viral vectors as they become more mainstream with an expectation that the F:E ratio and the active vs inactive particles are more fully understood and optimised. She added: “In other newer areas, e.g., iPSC derived products, there is a current lack of standardisation in, for example, cell line development which will develop and harmonise over time. eXmoor is optimising the DSP? and associated analytics for its AAV processes in the PD/AD labs to better characterise and optimise the products and regarding iPSCs is advising clients on cell line development in its consulting business assisting with briefing book preparation and leading discussions with regulators.”
TrakCel actively participates in various standardisation efforts including those led by the SCB to ensure regulatory alignment across the industry and we hope to see more changes supporting this. Ravi Nalliah, Chief of Product & Strategy of TrakCel, said: “The changes that ICCBBA made to the ISBT-128 standard around recommendations for the formatting of COI numbers are a good example of this. The reality is that any change within a regulated environment can be complex and time-consuming to implement. Our focus is on ensuring our product is structured so that it can by design be a facilitator rather than a blocker to change. The structure of OCELLOS ensures that new workflows and data validation rule can be implemented to help users navigate change and help sponsors to ensure mandatory regulatory criteria are met before a process can move on to the next step.”
The FDA has insisted that the potency assays become a focus and developers have put forth the effort to deliver, says Becky Cap, SVP, Business Development, Advanced Therapies at BioBridge Global, which will be critical for thinking in this area to become more nuanced and understand where a matrix of essays or multi parametric approach is the best solution for a particular cell type or disease state. She said: “At ISCT, we presented a poster describing our miniaturised dynamic potency assay. This is the critical path moving forward.”
