brittle bone disease News, Reviews & Resources

German biotech company Sirana Pharma GmbH has entered into a collaborative research agreement with Pfizer Inc. to investigate the potential identification and validation of a novel treatment concept for a rare bone disease, osteogenesis imperfecta (OI). Also called brittle bone disease, the condition is currently lacking approved drugs. Sirana Pharma focuses on the development of treatments for unmet medical needs in muscle and bone diseases. The collaboration will utilize Sirana’s microRNA (miRNA)-targeting approach, which targets substantial regenerative recovery of diseased muscle and bone tissue. “Combining Sirana’s novel miRNA-targeting approach, and the long-standing experience in musculoskeletal disorders and disease mechanisms of its founders, with the expertise and competencies of Pfizer will allow Sirana to expeditiously evaluate its drug candidates for the treatment of this rare disease and to identify potential candidates for further development,” said Michael Kring, CEO of Sirana Pharma GmbH. “At Pfizer, we are committed to advancing promising, emerging research – both through our internal capabilities and through collaborations with companies like Sirana – with the goal of bringing potentially life-changing medicines to patients around the world,” said Seng H. Cheng, senior vice president and chief scientific officer, Pfizer Rare Disease. “We look forward to working together with the Sirana team to further explore this new and innovative investigational approach, utilizing miRNA-targeting for the potential treatment of rare bone diseases.” Osteogenesis imperfecta Also known as brittle bone disease, osteogenesis imperfecta is a genetic disorder that causes weak bones that break easily in addition to other symptoms. There are several forms of OI, and although there is no cure, OI symptoms can be managed with a healthy lifestyle, medication, or surgery. According to the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the condition is a genetic or heritable disease in which bones break easily, often with no obvious cause or injury. Symptoms can range from mild with only a few fractures to severe with many medical complications. There are more than eight types of the disease, and those with a family history of it are at greater risk due to an abnormal gene passed on from one or both parents. Sirana Pharma, based in Munich, is focusing on the development of first-in-class molecules to treat sarcopenia – age related muscle weakness - and osteogenesis imperfecta – inherited brittle bone disease (an orphan drug indication).

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