Review: Research Xchange Forum 2018

The „Future Technologies” business area under the Pharma & Biotech division of Lonza has a focus on personalized medicine & cell therapy. One of the major challenges in these fields is the production of the patient-specific and patient-scale CAR-T cells or stem cells for treatment.

Bile duct disorders carry significant morbidity and mortality and remain a leading cause for liver transplantation in children and adults. Fotios invented a system for culturing bile duct organoids which reproduced key features of various biliary diseases in the lab.

Will’s initial research identified a new progenitor cell type within the inner ear, and showed that separate populations of progenitors can generate hearing, balance, and neural cell types. Will’s work demonstrated that a drug combination could induce the normally senescent cochlear hair cell progenitors to divide by reprogramming them to a more plastic state.

The body’s epithelial barriers are routinely exposed to damage causing pathogenic and noxious agents. Yet, our understanding of how barrier tissues cope with such recurrent pressures is limited. Naik found that the fundamental units of epithelial tissues, their stem cells, remember acute inflammatory encounters.

Kole developed a novel class of synthetic receptors called synthetic Notch (synNotch) receptors that directly induce a custom transcriptional response when they recognize disease or tissue-related cues.

The biopharmaceutical company apceth is harnessing the potential of mesenchymal stem cells (MSC), in which the mechanism of therapeutic effects are very different from that of CAR-T cells. The aim is to provide innovative cell-based gene therapies for the treatment of major indications, such as cancer, chronic lung and kidney diseases and for immunomodulation. 

Servier is applying the Cellectis’ TALEN gene editing technology to overcome current limitations and provide an allogeneic, frozen, “off-the-shelf” T cell based medicinal product.
UCART19 is an allogeneic CAR T-cell product candidate being developed for treatment of CD19-expressing hematological malignancies.

The generation of patient specific specialized cell-types is a major task in regenerative medicine. Despite the success of genetic reprogramming protocols, it is still a challenge to generate, cultivate and expand patient specific induced pluripotent stem cells (iPSCs).