Blueprint Medicines to Evaluate Opportunities to Advance Rare Disease Discovery Program in Fibrodysplasia Ossificans Progressiva Following Discontinuation of Collaboration with Alexion
"We believe our discovery research program in fibrodysplasia ossificans progressiva represents a potential opportunity to improve the understanding and treatment of this severe rare disease, and we intend to evaluate options to advance preclinical activities within our disciplined portfolio strategy," said
Pending its evaluation of opportunities to advance this program,
About Fibrodysplasia Ossificans Progressiva
FOP is a heritable, ultra-rare disorder of the connective tissue that is characterized by the abnormal transformation of skeletal muscle, ligaments and tendons into bone. As the disease progresses, the extra-skeletal bone increasingly restricts joints and other parts of the body. This inevitably leads to severe disability and breathing difficulties due to restricted chest wall expansion, which can be fatal in some instances. Currently, approved therapies are limited to ameliorating disease symptoms through the administration of steroids.
Blueprint Medicines is developing a new generation of targeted and potent kinase medicines to improve the lives of patients with genomically defined diseases. Its approach is rooted in a deep understanding of the genetic blueprint of cancer and other diseases driven by the abnormal activation of kinases. Blueprint Medicines is advancing four programs in clinical development for subsets of patients with gastrointestinal stromal tumors, hepatocellular carcinoma, systemic mastocytosis, non-small cell lung cancer, medullary thyroid cancer and other advanced solid tumors, as well as multiple programs in research and preclinical development. For more information, please visit www.blueprintmedicines.com.
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Kristin Hodous, 617-714-6674, firstname.lastname@example.org; Jim Baker, 617-844-8236, email@example.com