NascaCell GmbH, a German start-up founded in 2000, is using aptamer technology to gain new perspectives on drug discovery. The company uses its RNA-based Nucleic Acid Biotool robotics platform to combine target validation and small molecule screening to speed up drug discovery.
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For such an apparently well-understood molecule, it's certainly taken enough time for RNA to become a focus of drug discovery. But increasingly, it's the new, hot target. Companies focusing on or around RNA are multiplying. One of the best funded of the new companies is PTC Therapeutics, with $56 million raised so far. PTC is creating a platform around the post-transcriptional modulation of RNA, aiming to increase or decrease gene expression by tinkering with the mechanics of how RNA makes proteins.
There's growing conviction that gene arrays are less useful than first imagined for expression studies and diagnostics--and that directly reading protein expression will more likely provide an accurate picture of biological status-health, disease, and pharmaceutical response. Thus, a number of companies have started up to create, on the analogy of gene arrays, protein arrays, to allow the simultaneous and quantitative testing of large numbers of proteins-potentially thousands.But the task is easier said than done. First, there are too few proteins known to allow testing for worthwhile expression patterns; and, second, because of the delicacy and variety of proteins, surface chemistry and other problems have historically made arrays impractical. None of this daunts the start-ups who, opportunistic, are looking to exploit the markets they think they can get to first: clinical diagnostics and pharmaceutical clinical development programs.
Recent excitement around exosomes underscores their potential to solve drug delivery challenges that have limited the power and applicability of the biopharma industry’s growing arsenal of therapeutic modalities.