Two members of our newly established Advanced Therapies business unit; Dr Germano Ferrari and Dr Silvana Bardelli, attended the World Advanced Therapies and Regenerative Medicine Congress 2019 in London. We caught up with Germano to get his thoughts on the three-day event.
Having attended the congress multiple times over the past five years, it’s clear from the increasing number of participants that there is a growing interest in specific technologies – particularly CAR-T, which shows great promise in fighting cancer.
Participants were not limited to the likes of academics and start-ups, who are focused on the core science, but spanned the whole eco-system of international service providers and investors. The inclusivity of the congress underlined the importance of collaboration across the board, in order to find the most efficient and financially viable way to bring cell and gene therapies to patients globally.
The agenda spanned a variety of topical discussions and therapy areas, so whilst I attended some sessions aligning to my interest in new technologies and therapies in development, Silvana attended sessions around her own areas of interest: regulatory and patient engagement in the clinical setting. A highlight of the conference was the keynote lecture delivered by Nobel Prize winner Prof Shinya Yamanaka on induced Pluripotent Stem Cells (iPSCs) research and application.
Hot Topics – therapeutic approaches:
CAR-T therapies: an entire universe of companies is following the success of Kite and Novartis, supported by an increasing community of specialised investors.
Product expansion: current CAR-Ts are focused on CD19 receptor. Scientific data suggest that the same technology could be used with other receptors, potentially extending the number of diseases treated;
Safety: there is still a major discussion concerning safety issues, specifically cytokine release syndrome (CRS) and neurotoxicity which occur with all CAR-T products currently available.
Gene editing: after 1,000 scientific articles published, it is currently undergoing heavy ethical discussion following the recent Chinese experiments using CRISPR technology on human embryos to generate kids immune to HIV. Scientific concerns are around the improvement of accuracy of sequence targeting which is linked to safety and the clinical translation of this approach. This issue has not been resolved yet.
Cord blood banks: while their capacity is saturated and it is not very clear how to use the cells already banked, there is still great hope to find a proper and efficient way to apply them.
EASY and ELEGANT: at least two cases of “simple” cell therapy approached were presented, receiving very favourable welcome :1) Holoclar (from Holostem , Chiesi) – a cell therapy for the treatment of limbal stem-cell deficiency (on the market), and 2) Caladrius CD34 therapy (CD34+ cells coming from the patient blood stream are concentrated and injected locally to repair heart and limb ischemia).
The MAKE vs BUY conundrum:
While there is limited capacity available in Europe for clinical/market grade manufacturing of advanced therapies, companies are still debating whether to build their own manufacturing facilities or to externalise the service to a few, international providers with relevant experience. The discussion is ongoing and only in the next few years, with a lot of potential new launches on the market, will we be able to have an answer.
Treat larger numbers of patients:
As the key requirement for therapies moving from the development stage to the market, across the board there are currently deep discussions around the possibility to produce in larger volumes through use of improved manufacturing processes and elements of automation.
One of the main aspects discussed is the possibility to change the current products in development to make them available to a larger number of patients by transforming “autologous” products (= cells coming from a patient are modified and reintroduced into the same patient) into “allogeneic” products (=cells are coming from external sources where the same batch of cells can be used to treat multiple patients). This looks set to be one of the primary challenges for the next few years.
It is certainly an exciting time to be working in this increasingly innovative field. However, whilst there are several providers that have been involved in the development of advanced therapies in the past few years (such as viral vector manufacturers), many major bottle necks in this field are still present and need to be addressed to enable advanced therapies to be used widely with patients. I expect most of these will likely remain the focal topics of discussion for years to come.
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