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It also develops therapeutics in the areas of infectious disease, rare liver disease, immuno-oncology, and cardiovascular disease. Its pipeline includes various drug modalities such as prophylactic vaccines, therapeutic vaccines, intratumoral immuno-oncology, localized therapeutics, and liver intracellular.
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R&D grant funding initiative for collaborative projects with research centers and early start-ups across North America that explore emerging science and technologies (e.g. digital and data science, high-throughput screening, drug delivery, and imaging tools) that can be applied across the therapeutic pipeline.
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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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Opportunity to join a best-in-class team to support a proven therapeutic in oncology rare disease and ready the marketplace for a launch brand. Enabled by our proprietary drug discovery platform, Deciphera has developed a diverse pipeline of wholly-owned drug candidates.
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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 vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. Experience working in rare disease is desirable, but not required.
$156,000 - $195,000 a yearFull-timeExpandApply NowActive JobUpdated 2 days ago - UpvoteDownvoteShare Job
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They will be motivated by the opportunity to bring a rare disease commercialization mindset and to build their markets which will lay the foundation for the success of Pyrukynd and Agios’ pipeline of transformative rare genetic disease treatments.
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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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We are looking for candidates with deep experience (20+ years) in the healthcare industry whose background includes working with different rare diseases and gene therapy products. Evaluate localized health policy and health care reform trends, assessing implications to the Intellia Therapeutics pipeline and portfolio.
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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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rare pipeline jobs in Cambridge, MA
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