Shire plc, theglobal specialty biopharmaceutical company, announces today that it hasentered into a research-based collaboration with Santaris Pharma, aleading player in RNA-based therapeutics, to develop its proprietaryLocked Nucleic Acid (LNA) technology in a range of rare diseases,thereby enabling Shire to build on its already strong competitiveposition for its Human Genetic Therapies (HGT) business. LNA technologyhas the benefit of a very quick validated target to proof of conceptturnaround, thereby increasing the speed and lowering the cost ofdevelopment.
Currently, Shire HGT has a unique and successfulplatform producing human cell derived enzyme replacement therapies(ERTs) for a wide range of rare human genetic disorders.
SylvieGrégoire, President of Shire HGT, comments, “With this novel platformtechnology Shire has the potential to move into a wider range ofgenetic orphan diseases. We will look to develop treatments for peoplewith a variety of rare life-altering and life-threatening conditionsthat to date, could not be effectively addressed by enzyme replacementtherapy.”
As part of the joint research project SantarisPharma will design, develop and deliver pre-clinical LNAoligonucleotides specific for the selected Shire targets, and Shirewill have the right to further develop and commercialize thesecandidate compounds on a worldwide basis.