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Evidence of being a critical strategic thinker who is solution oriented with the ability to think outside of the box; preference for demonstration of such mindset within rare disease programs.
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Experience working in rare disease is desirable, but not required. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.
$156,000 - $195,000 a yearFull-timeExpandApply NowActive JobUpdated 2 days ago - UpvoteDownvoteShare Job
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Oral oncolytic or rare disease experience, preferably including launch experience. Enabled by our proprietary drug discovery platform, Deciphera has developed a diverse pipeline of wholly-owned drug candidates.
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It also develops therapeutics in the areas of infectious disease, rare liver disease, immuno-oncology, and cardiovascular disease. Ensure the medical accuracy of a broad spectrum of materials, from disease awareness and pipeline communications to marketing and internal documents.
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Provide strategic guidance for and author applications related to expedited programs/designations for rare muscle disease (e.g. Fast Track, Orphan Drug Designation, etc) Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD.
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Our diversified pipeline includes clinical programs in Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington’s disease, as well as a preclinical program in obesity. Our RNA medicines platform, PRISM, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders.
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We are supporting a leading biotechnology company in rare disease and neurology development as they continue to expand their already healthy pipeline. This organization are at a pivotal stage of growth and already have one product on the market as well as an exciting pipeline of products.
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This person will report to the Director, Scientific Communications for Rare Disease, Pipeline, and Neuropsychiatry and will have the opportunity to work in multiple therapeutic areas, primarily leading publications.
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This is a rare opportunity to join a close-knit team supporting a proven oncology therapeutic while preparing the marketplace for a launch brand in rare disease and other pipeline assets.
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Vertex currently operates at the forefront of rare disease scientific innovation and has successfully developed and commercialized multiple breakthrough medicines for Cystic Fibrosis (CF) and Sickle Cell Disease.
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AU-011, our first VDC candidate, is being developed for the first line treatment of primary choroidal melanoma, a rare disease with no drugs approved. Responsibility for overall pipeline and portfolio delivery in Ocular Oncology, working with Preclinical, Regulatory Affairs, Quality, Clinical Operations, and other relevant departments and functions.
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Our drug discovery efforts to target switch sites has rapidly advanced, resulting in an emerging pipeline of research programs that address well-validated, genetically defined transcription factors, initially focused on precision oncology with future potential in neurology, rare genetic disorders, immunology and inflammation, and obesity.
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The Senior Manager, Scientific Communications is a member of the Global Scientific Capabilities department within Medical Affairs and is responsible for the strategic planning and execution of high-quality scientific communications deliverables.
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Building on the company’s leadership in the field of cellular metabolism, Agios is advancing a robust clinical pipeline of investigational medicines with active and planned programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia.
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Oversee our relationship with the Rare Disease Company Coalition. This role will play a key role in the evolution of our organization and lead the patient affairs strategy throughout the continuum of preclinical and clinical development to commercialization for Astria’s pipeline of programs.
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rare disease pipeline jobs in Waltham, MA
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