Rational's molecular delivery technology platforms can enable a broad spectrum of medical research and therapeutic development. Rational has ongoing programs in the following arenas:

Predictive Drug Discovery Platform

High throughput cell-based assay technologies make it realistic to perform large-scale functional assays for revealing pharmacological roles of genes, proteins and pathways in complex cellular context. Human primary cells preserve many important in vivo characteristic cellular functions and processes, therefore, they are the best in vitro models to the human body.

Rational is in development of platforms that evaluate functional activities of certain classes of proteins, such as transcription factors, in human primary cell cultures under influence of drugs. Through interrogating and interpreting these large amount of functional data, Rational strives to build predictive models that offer more accurate prediction of a drug's side-effect and treatment efficacy, and moreover, provide guidance on how to engineer drugs to achieve desirable therapeutic effects.

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Liver Therapy

Liver fibrosis and cirrhosis are major diseases with high mortality rates. Although currently no therapies show effectiveness in cure these formidable diseases, studies have shown that intervention of certain cellular pathways could lead to stop progression, or even reverse fibrotic build up in liver.

Rational explores the cutting-edge RNAi technology in treating alcoholic liver, liver fibrosis and cirrhosis. Effective and selective delivery of siRNA agents into liver, which is one of the major bottlenecks in RNAi therapy, will be realized by our patent-pending delivery technology, which have demonstrated its superior capability in in vitro tests. Rational strives to facilitate development of minimally invasive technology for treating patients with these liver diseases.

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CNS Drug Discovery

Central Nerve System (CNS) disorders and diseases, such as Alzheimer's disease, Parkinson's diseases and brain tumor, are among the most prevalent and formidable diseases that remain to be intercepted by medicine. One of the major challenges in CNS drug development is to overcome the blood-brain-barrier (BBB), brain's protective mechanism that excludes most compounds from enter the brain. A variety of transporter proteins are highly expressed in BBB and are responsible for preventing drugs from crossing BBB, while in meantime facilitating transport of various nutritious molecules into the brain.

Rational's revolutionary transporter assay technology permits high-throughput screening of compounds that interact with BBB specific transporters. Rational's team designs drugs to utilize these transporters for effective BBB penetration. Rational is developing screening assays for selecting compounds with indications for treating brain tumor and CNS inflammation.


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