RNA interference is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a protein of interest. Mediated by small interfering RNAs (siRNA), a class of RNA (ribonucleic acid) molecules, 20-25 nucleotides in length, RNAi-based therapeutics can leverage this natural pathway of gene silencing to potentially target and shut down specific disease causing genes.
Conceptually, RNA interference allows silencing specifically any gene. In degenerative diseases, where particular genes have an unregulated activity, siRNA targeting of the specific gene can silence its activity and control the disease. Following the 2001 discovery of RNAi in human cells, it was widely acclaimed as opening up a new era in drug development for degenerative diseases, with a broader therapeutic potential and greater target specificity than small‐molecule drugs.
Since discovery of the RNAi mechanism in humans, siRNA‐based therapies bear great promise because of the ability to design precise RNA strands that target specific proteins that command cell activity. They could be the ultimate response to cancer and other degenerative diseases, since they allow modulating treatment to a precise protein in the cell.
Advantages of siRNA therapies include better specificity (the ability to target for almost any gene, regardless of the function of the gene product), high potency (RNAi‐mediated inhibition is more potent than that achieved with antisense oligonucleotides) and versatility (due to its superior potency, it is also relatively easy to identify effective RNAi target sites).
Certain drug targets such as intranuclear genes and some proteins are difficult to inhibit with traditional drug-based and biologic therapeutics. Developing effective drugs for these targets would have the potential to address a very large underserved market for the treatment of many diseases. Using its potential to specifically target and silence virtually any gene target, siRNA therapeutics may be able to address previously "undruggable" targets, unlocking the market potential of such targets.
Phyzat’s discovery and clinical development programs are based on proprietary siRNA technology.
We actively seek collaboration and licensing agreements with leading biopharmaceutical companies to advance the development and commercialization of our technology platforms and drug candidates.
We expect to access external expertise and capital to complement our internal research and create commercialization opportunities in areas outside of our core focus.
We have established collaborations with the following entities:
Center for Drug Discovery and Innovative Medicines, University Porto
Marks & Clerk
Charles River Discovery Research Services Germany GmbH (formerly, Oncotest)
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