How do we create an economic model to put stem cell products on the market? From the industrial point of view, the answer can be simply down to the inversely proportional relation when to decreasing CoGs correspond increasing company revenues.
An example of scalable manufacture and distribution strategy resulted from a successful collaboration between science and industry was demonstrated by the case study of joint scale up manufacture achieved by Promethera Bioscience and Pall Life Sciences.
Promethera was born in 2009 as a spin-off of Belgian UCL (Université Catholique de Louvain) and back then it employed only 2 people. Today their staff has grown to almost 50 employees and it holds a pivotal role in the development and commercialization of cell therapies for the treatment of liver diseases using allogeneic adult progenitor hepatocytes isolated from healthy livers.
Promethera’s pipeline focuses on the generation of two products: HepaStem and Hepa2/3Screen (the latter consisting in optimized cell based assays). HepaStem is their main one and it is processed for liver based-metabolic diseases and acquired liver disorders, and has recently reached phase IIb for Crigler-Najjar and urea cycle conditions. It is allogenic, world-wide distributed, can can be formulated on-site and also easily reconstituted at the bedside of the patient by the medical team itself.
After reaching a full development stage and once comparative studies for in process, quality and release control were performed, HepaStem success was enabled by up-scaling from a multitray stack system to Pall’s Xpansion bioreactor system, which allowed a 13-fold scale up of p5 liver progenitor cells.
Since October 2013, 4 full-scale batches were produced for phase IIb clinical trials around the world by using only 5 XP200 bioreactors of the Xpansion platform family, and Xpansion bioreactors will also be used to facilitate the trials progression to phase III, while mimicking 2D cell growth, reducing footprint impact and maintaining the compact, closed and monitored bioprocessing conditions.
With regards to future perspectives, going commercial would need further upscaling technologies, and once again Pall will be able to provide the solution with the SoloHill microcarrier technology.
Sarah Snykers (Promethera Biosciences) and Dr José Castillo (Pall Life Sciences) told us their story, depicting a successful scenario on how the translation of a manufacturer’s needs into reliable products can lead to a win/win situation.
Giulia Detela for Total BioPharma at WSCRM Congress, London
Giulia Detela is a PhD Student from the Department of Biochemical Engineering, University College London.