Dicerna™ to Present at H.C. Wainwright 21st Annual Global Investment Conference
Sep 03, 2019
A live audio webcast of the presentation will be accessible within the Investors & Media section on the Dicerna website at www.dicerna.com. An archived replay of the webcast will be available on the Company’s website after the conference.
GalXC™ is a proprietary technology platform invented by Dicerna to discover and develop RNAi-based therapies designed to silence disease-driving genes in the liver. Compounds produced via GalXC are intended to be broadly applicable across multiple therapeutic areas involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases. Using GalXC, Dicerna scientists attach N-acetylgalactosamine sugars directly to the extended region of the Company’s proprietary RNAi molecules, yielding multiple proprietary conjugate delivery configurations. Many of the conjugates produced via GalXC incorporate a folded motif known as a tetraloop in the extended region. The tetraloop configuration, which is unique to Dicerna’s GalXC compounds, allows flexible and efficient conjugation to the targeting ligands and stabilizes the RNAi duplex, which the Company believes will enable subcutaneous delivery of its RNAi therapies to hepatocytes in the liver, where they are designed to specifically bind to receptors on target cells, potentially leading to internalization and access to the RNAi machinery within the cells. The technology may offer several distinct benefits, as suggested by strong preclinical data. The benefits seen in preclinical studies include: potency that is on par with or better than comparable platforms, highly specific binding to gene targets, long duration of action, and an infrequent subcutaneous dosing regimen.
Dicerna™, GalXC™, and PHYOX™ are trademarks of
Stern Investor Relations, Inc.
Lauren Stival, 212-362-1200
Alex Van Rees, 973-442-1555 ext. 111