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Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.
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Reporting to the Head of Marketing – Rare Disease, this role will be a highly visible position within the organization with a focus on the continued launch efforts in Friedreich Ataxia, maximizing its market potential, leading overall US strategy and execution.
Full-timeExpandApply NowActive JobUpdated 27 days ago - UpvoteDownvoteShare Job
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The Director of Cell Therapy will be a senior role in the Genomic Medicine & Cell Therapy organization in Alexion, AstraZeneca Rare Disease. The successful applicant will contribute to developing Alexion’s strategy for cell therapy of rare diseases through collaboration with the broader leadership in Research at Alexion and AstraZeneca.
Full-timeExpandApply NowActive JobUpdated 25 days ago - UpvoteDownvoteShare Job
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Experience in rare disease, pediatric, and/or complex clinical trials preferred. The CPM will be responsible for all aspects of clinical trial management aimed at treating a rare or infectious liver-targeted disease.
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Clinical experience within neuromuscular disorders (e.g., DMD, DM1) preferred; rare disease experience considered. Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases.
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This will involve the application of advanced computational approaches in statistical genetics and predictive modeling (e.g. polygenic modeling, rare variant burden tests, disease trajectory analyses) to biobank and other human genetics datasets.
Full-timeExpandApply NowActive JobUpdated 26 days ago - UpvoteDownvoteShare Job
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Specialty Pharmacies & Specialty Distributors : Understanding of the operations and role of SPs/SDs in managing the journey of patients with rare disease. Presentations : Deliver presentations on disease state, clinical, economic, access, and business topics effectively.
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Experience with rare disease and/or pediatric trials also preferred. Scientifically and clinically agile, proven ability to learn and apply relevant disease information into strategic operational planning and delivery.
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Experience in rare disease strongly preferred. Alnylam Pharmaceuticals (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and more prevalent diseases.
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As the TA Partner, GMSGQ Biologics, you’ll partner with managers to find great talent for our Biologics Operating Unit, a network of manufacturing sites producing cell-based therapies that treat many rare diseases across the franchises of Internal Medicine, Genetic Disease, Gastroenterology, and Hematology.
$170,500 a yearFull-timeExpandApply NowActive JobUpdated 21 days ago - UpvoteDownvoteShare Job
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A minimum of 3-5 years of experience in the pharmaceutical/biotech industry and 1-2 years in patient advocacy preferred; particularly with rare disease patient populations. A minimum of 3-5 years of experience in the pharmaceutical/biotech industry and 1-2 years in patient advocacy preferred; particularly with rare disease patient populations.
Full-timeExpandApply NowActive JobUpdated 28 days ago - UpvoteDownvoteShare Job
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Therapeutic Area : Rare Disease. Therapeutic Area : Rare Disease. Required: BS or MS in a relevant field with a minimum of 3 years’ experience, specializing in UPLC/HPLC analytics and LNP formulation.
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Sanofi Rare and Neurological Disease (RND) research therapeutic area is recruiting for a total of 6 co-op or interns for 2025 at its US Cambridge Crossing site. 2025 Co-op - Sanofi Rare and Neurological Disease (RND) page is loaded.
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Making a positive impact to help ultra-rare disease patients who are in need of life-saving treatments. We are dedicated to developing and delivering innovative therapies to improve the lives of people who live with a rare disease.
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The group is known for its products in key therapeutic areas such as Women's Health, Neurology and Psychiatry, Cardiovascular and Metabolic, and Rare Disease. A proven track record of identifying, initiating, developing, and maintaining strategic business relationships with HCPs in a rare disease space.
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rare disease jobs in Lexington, MA
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