Gene Targeting and RNA - based Therapeutics
Combining next generation of nucleic acid - based therapeutics and an innovative novel technology platform
Designing and Delivering the Next Generation Nucleic Acid Therapeutics
Delivering the next generation of nucleic acid therapeutics is only one component of our broad strategy. Once you design a new therapy, you have to deliver it to the disease target site. In fact, many times in drug discovery that is the most difficult part of the problem. By leveraging our library of novel new class of nanoparticles and innovative proprietary platform technology, we are developing first in class therapies for central nervous system disorders.
RNA therapeutics are a new class of RNA based therapies and therapeutic agents. RNA is central to all biological processes. Therefore, focusing on RNA as a therapeutic target has become a major avenue of research and discovery. There are many potential avenues for addressing human diseases and disorders at the RNA level. At SMDG, we leverage our novel platform technology and library of new class of nanoparticles combined with the next generation nucleic acid therapies designed within our discovery programmes to deliver life saving therapies.
Ribonucleic acid (RNA) is a molecule that has significant and essential roles in biological processes including in coding of genetic information, participating in decoding, functioning in regulation and expression of genes. RNA and DNA (Deoxy-ribonucleic acid) are both nucleic acids. Along with lipids, proteins, and carbohydrates, nucleic acids constitute one of the four major classes of macromolecules essential for all known forms of life. Like DNA, RNA is assembled as a chain of nucleotides. However, unlike double - stranded DNA, RNA is usually found in nature as a single-stranded molecule that forms internal base pairs as it forms itself into a 3-D structure. Cellular organisms use messenger RNA (mRNA) to convey genetic information (using the nitrogenous bases of guanine, uracil, adenine, and cytosine, denoted by the letters G, U, A, and C) that directs synthesis of specific proteins.