Shire and Santaris extend rare genetic disease pact

Strategic alliance will continue to discover and develop LNA-drugs in the rare genetic disease field
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HOERSHOLM, Denmark and SAN DIEGO—Santaris PharmaA/S, a clinical-stage biopharmaceutical company focused on the discovery anddevelopment of RNA-targeted therapies through its Locked Nucleic Acid (LNA)Drug Platform, recently announced that its long term partner, Shire, hasextended their existing partnership in the rare genetic disease space.
Under the terms of the extended agreement, Shirewill have the right to nominate additional collaboration targets for drugdiscovery and development. Santaris will receive an upfront payment and,consistent with the initial agreement, is eligible for research support, preclinical,clinical and sales milestones and royalties on each product emerging from thecollaboration.
"Our collaboration with Shire is veryimportant to us so we are very pleased with the decision to extend theagreement and allow for more drug discovery and development programs," said HenrikStage, president and CEO at Santaris Pharma. "We believe the LNA drug platformoffers a unique opportunity to develop drugs against the rapidly expandingnumber of disease targets in the rare genetic disorder space."
"We are delighted with our partnership withSantaris, whose expertise and capabilities in LNA antisense therapy complementShire's drug discovery and development strengths," added Albert Seymour, vice presidentof discovery research at Shire. "Our hope is that the partnership willeventually translate into novel drugs that will help patients suffering fromdebilitating rare diseases lead better lives."
The LNA drug platform and drug discovery enginedeveloped by Santaris Pharma combines the company's proprietary LNA chemistrywith its highly specialized and targeted drug development capabilities torapidly deliver LNA-based drug candidates against RNA targets, both mRNA andmicroRNA, for a range of diseases including cardiometabolic disorders,infectious and inflammatory diseases, cancer and rare genetic disorders. LNA isalso sometimes referred to as BNA (bicyclic or bridged nucleic acid).
Santaris describes LNA-based drugs as "a promisingnew class of therapeutics that are enabling scientists to develop drugcandidates to work through previously inaccessible clinical pathways," noting thatits LNA Drug Platform "overcomes the limitations of earlier antisense and siRNAtechnologies to deliver potent single-stranded LNA-based drug candidates acrossa multitude of disease states. The unique combination of small size and veryhigh affinity allows this new class of drugs candidates to potently andspecifically inhibit RNA targets in many different tissues without the need forcomplex delivery vehicles."

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