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Shire Builds Competitive Position of its HGT Business through Research Collaboration with Santaris Pharma A/S on LNA Drug Platform

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. The Company's own research and development activities focus on infectious diseases and metabolic disorders while partnerships with major pharmaceutical companies include a range of therapeutic areas including cancer, rare genetic disorders and inflammatory diseases. Santaris Pharma A/S, founded in 2003, is headquartered in Denmark with operations in the US. The Company has strategic partnerships with Enzon Pharmaceuticals, GlaxoSmithKline, Wyeth Pharmaceuticals and Shire plc. Please visit for more information.

Notes to editors


Shire's strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder (ADHD), human genetic therapies (HGT) and gastrointestinal (GI) diseases as well as opportunities in other therapeutic areas to the extent they arise through acquisitions. Shire's in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights. Shire believes that a carefully selected and balanced portfolio of products with strategically aligned and relatively small-scale sales forces will deliver strong results.

For further information on Shire, please visit the Company's website:


Statements included herein that are not historical facts are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, the Company's results could be materially adversely affected. The risks and uncertainties include, but are not limited to, risks associated with: the inherent uncertainty of research, development, approval, reimbursement, manufacturing and commercialization of the Company's Specialty Pharmaceutical and Human Genetic Therapies products, as well as the ability to secure and integrate new products for commercialization and/or development; government regulation of the Company's products; the Company's ability to manufacture its products in sufficient quantities to meet demand; the impact of competitive therapies on the Company's products; the Company's ability to register, maintain and enforce patents and other intellectual property rights relating to its products; the Company's ability to obtain and maintain government and other third-party reimbursement for its products; and other risks and uncertainties detailed from time to time in the Company's filings with the Securities and Exchange Commission.

    For further information please contact:

    Media          Jessica Cotrone            +1 617 613 4640

SOURCE Shire plc
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