The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Genespire have published data highlighting progress in the development of an improved targeted gene replacement technology in human hematopoietic stem cells (HSCs) in Nature Biotechnology.
Human stem cells were once viewed primarily as regenerative materials for tissue repair through cell therapies. However, advances in technologies and protocols mean that induced pluripotent stem cells (iPSCs) are now playing an increasingly important role in disease modelling and human cell-based screening assays. This article explores what this means for drug discovery.
This article discusses large-scale generation of patient-derived induced pluripotent stem cells (iPSCs) to accelerate the understanding of disease biology and drug discovery.
Recently, pre-clinical and clinical studies of stem cells in regenerative medicine have demonstrated great promise as treatments for various diseases. Their ability to self-renew and differentiate makes stem cells valuable for both cell therapy and tissue engineering fields, both of which drive the demand for largescale cell expansion.
A recent market survey on stem cells in research and drug discovery showed that despite a significant amount of hype and hope around stem cells, most drug discovery-related efforts today still fall into the category of basic research and the majority of that was directed towards the oncology/cancer disease area.
With the failure rates of drug candidates continuing to present phenomenal costs to the pharmaceutical industry, this article discusses how newly emerging induced pluripotent stem cell (iPSC) technologies have the potential to be an effective tool in weeding out low quality candidates early in the process, reducing attrition costs and, ultimately, improving the percentage of new drugs to market.
Stem cells remain a hot topic in academia and industry alike, and with the potential to cause a paradigm shift where many believe in their ability to differentiate into a variety of valuable cell types.
While the jury is still out in terms of iPS cell technology and its ability to deliver a regenerative therapy or, indeed, contribute to the development of a clinical molecule, this paper argues that the progress by which this technology has been advancing is providing confidence that it will become a breakthrough technology for drug discovery.
The area of cellular therapeutics has never been readily embraced by 'Big Pharma', with ethical and regulatory issues even more complex and troublesome than proteins. Can stem cell therapy provide the key to unlocking a rich vein of pharmaceutical activity?
Induced pluripotent stem (iPS) cells have the potential to transform drug discovery by providing physiologically relevant cells for toxic compound identification, target validation, compound screening, and tool discovery. The technology for generating iPS cells is advancing rapidly, as is the repertoire of cell types that can be differentiated.
This article explores the emerging market landscape vis--vis the use of stem cells as cellular therapeutics. For more than 40 years, hematopoietic stem cells have been utilised as cellular therapeutics in a number of malignant diseases as well as inborn genetic errors of metabolism.
Over the past decade, the use of cell-based assays has accelerated in modern drug discovery. Indeed, the majority of assays in either target validation or lead identification/optimisation all now employ cell-based technologies.