CAMBRIDGE, Mass., Aug 24 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces today that it has entered into a research-based collaboration with Santaris Pharma A/S, a leading player in RNA-based therapeutics, to develop its proprietary Locked Nucleic Acid (LNA) technology in a range of rare diseases, thereby enabling Shire to build on its already strong competitive position for its Human Genetic Therapies (HGT) business. LNA technology has the benefit of a very quick validated target to proof of concept turnaround, thereby increasing the speed and lowering the cost of development.
Currently, Shire HGT has a unique and successful platform producing human cell derived enzyme replacement therapies (ERTs) for a wide range of rare human genetic disorders.
Sylvie Gregoire, President of Shire HGT, comments, "With this novel platform technology Shire has the potential to move into a wider range of genetic orphan diseases. We will look to develop treatments for people with a variety of rare life-altering and life-threatening conditions that to date, could not be effectively addressed by enzyme replacement therapy."
As part of the joint research project Santaris Pharma A/S will design, develop and deliver pre-clinical LNA oligonucleotides specific for the selected Shire targets, and Shire will have the right to further develop and commercialize these candidate compounds on a worldwide basis.
About Locked Nucleic Acid (LNA) Drug Platform
The Locked Nucleic Acid (LNA) Drug Platform developed by Santaris Pharma A/S creates synthetic chemical versions called LNAs of the normal nucleic acid building blocks of ribonucleic acids (RNA). These LNAs improve the drug-like qualities of resulting therapeutics, called oligonucleotides, by boosting resistance to metabolism, increasing half-life and improving tissue uptake. LNA-based therapeutics demonstrate improved binding affinity to their target RNA, which increases potency many-fold over other nucleotide therapeutics. The greater potency of LNA in binding complementary RNA sequences also allows for the use of significantly shorter LNA oligonucleotide drugs which can be more effective than previous antisense or siRNA drugs.
About Santaris Pharma A/S
Santaris Pharma A/S is a privately-held biopharmaceutical company focused on developing RNA-based drugs targeted to disease-related mRNAs and microRNAs. Utilizing the Company's proprietary Locked Nucleic Acid (LNA) Drug Platform and Drug Discovery Engine, Santaris Pharma A/S provides fast and efficient generation of lead LNA drug candidates.
