Our Science & Philosophy
In the latter part of the 20th century, the pharmaceutical industry witnessed significant advancements by developing drugs such as cholesterol-lowering agents, HIV protease inhibitors, and targeted cancer medications. This progress underscored a systematic approach encompassing the elucidation of pivotal biological pathways in disease pathogenesis, the establishment of high-throughput screening assays for drug discovery, preclinical testing in animal models, medicinal chemistry-based optimization of lead compounds, and validation of safety and efficacy through clinical trials to meet regulatory approval prerequisites before commercialization
Yet, many setbacks in late-stage clinical trials have hindered innovation, resulting in a predominant trend towards derivatives, generics, and biosimilars rather than pioneering pharmaceutical introductions. Furthermore, the evolving medical landscape, characterized by chronic and poorly understood diseases, has incurred substantial societal costs
Hence, there is a compelling need for a revised drug discovery framework emphasizing understanding disease natural histories and identifying novel therapeutic targets. This redesigned paradigm must inevitably prioritize patient-centric considerations from inception through to fruition
In vitro and ex vivo methodologies are pivotal for enhancing our comprehension of human biology and the mechanisms underlying diseases. In vitro experiments are conducted in a controlled setting outside of living organisms, commonly utilizing test tubes or petri dishes. In contrast, ex vivo studies focus on cells or tissues extracted from living organisms and maintained in artificial environments, offering a window into biological processes that is more reflective of physiological conditions than traditional in vitro systems. These platforms, particularly when leveraging primary and progenitor human cells or tissues, yield findings more translatable to human health than conventional animal models or immortalized cell lines
Researchers have advanced the growth of virtually all human cell types in laboratory settings, including developing 3D structures that mimic human organ systems through induced pluripotent stem cell technology. This innovation provides a more physiologically relevant framework for testing novel therapies. Cell culture techniques have been instrumental in significant advances across various domains: oncology, sepsis, nephrology, and HIV/AIDS, along with routine applications in chemical safety assessments, vaccine production, and drug discovery
Utilizing both healthy and diseased human tissues from volunteer donations presents a more pertinent method for studying human biology and disease pathology than relying on animal models. Human tissues can be sourced from well-characterized biological discards, which are readily available. The increasing capabilities of computational models enable researchers to replicate aspects of human physiology, bolstered by data from relevant in vitro or ex vivo platforms that can inform integrated predictive models
In oncology research, the focus is on drug discovery—either via novel agents or repurposing existing ones—that targets complex and metastatic cancers. This is achieved by manipulating diverse stem cells sourced from donors and patients, conducting drug screening on patient-derived samples, and developing predictive platforms replicating the tumour microenvironment using patient-derived materials. This next-generation approach allows examining previously considered untreatable tumours and “undruggable” molecular pathways, such as Wnt, Ras, and cMyc. Tumour-initiating cells, which reside within neoplastic tissue and give rise to malignant phenotypes, are central to tumorigenesis, emphasizing the urgent need to explore targeted pharmaceutical interventions that selectively eliminate these stem cell populations
Exploring therapeutics for neuromuscular and rare diseases through preclinical research involving in vitro and ex vivo platforms, particularly those incorporating human microphysiologies, can generate critical clinical insights that address the limitations of traditional animal or transformation-based models