Access Pharmaceuticals reports significant progress in Cobalamin-targeted drug-delivery program for siRNA therapies

August 01, 2017

RNAi is typically initiated by the introduction of small fragments of RNA, termed siRNA, into cells at disease sites. Due to their large size and high negative charge, siRNA fragments are not able to cross cell membranes. Therefore, to develop effective RNAi therapeutics, a delivery system must be developed that can transport the siRNA into cells, and release undamaged siRNA into target cell cytoplasm.  The CobaCyte technology is particularly well-suited for this purpose.  Most human cells have a requirement for vitamin B12 which is served by cell surface receptors which facilitate absorption of this vitamin.  In many diseases, the demand for vitamin B12 is increased, with a corresponding upregulation of the receptor. Using the 'Trojan Horse' principle, the CobaCyte nanoparticle technology can utilize the vitamin B12 uptake mechanism to transport siRNA into cells whereupon native siRNA can be released for incorporation in messenger RNA (mRNA) to initiate the beneficial therapeutic effect. In this way, CobaCyte offers the potential for targeted delivery of siRNA. The fact that Access' vitamin B12 technology also facilitates oral drug delivery indicates that it may be possible for this technology to provide effective siRNA treatments by oral drug delivery.

For additional information on CobaCyte siRNA please click on the following link to view Dr. Nowotnik's recent invited presentation at the Novel Nanodelivery of siRNA and miRNA Symposium, hosted by the California NanoSystems Institute at UCLA: accesspharma/downloads/presentations/Cobalamin-Slides.pdf

SOURCE Access Pharmaceuticals, Inc.