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Experience contributing the design and implementation of natural history studies and registries in the pharmaceutical industry is required; experience with rare disease is a plus. Contribute to the development of real world evidence strategies for post marketing programs, and health economics and patient outcomes research initiatives.
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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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Company OverviewSince its inception over a decade ago, CRISPR Therapeutics has transformed from a research-stage company advancing programs in the field of gene editing, to a company with a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular and rare diseases.
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Experience in Rare Disease Drug Development and familiarity with gene and/or cell therapy preferred. Reporting to the VP of Biometrics, the Director/Sr. Director, Clinical Data Management is responsible for all clinical data management activities related to the effective planning and timely delivery of complete, high quality and reliable clinical trial data.
Full-timeExpandApply NowActive JobUpdated 23 days ago - UpvoteDownvoteShare Job
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Prior experience in rare disease drug development is highly desirable. We are seeking a visionary leader with deep expertise in data analytics, a proven track record of driving innovation, and a passion for advancing rare disease research.
ExpandApply NowActive JobUpdated 20 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 role reports to the Head of Marketing, Rare Disease. The Senior Product Manager, HCP Marketing plays a major role in the design, development and execution of marketing strategies for assigned product(s) within the Rare Disease franchise with an emphasis on HCPs.
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Join Takeda as a Lead Data Engineer RDBU where you will help shape and implement the vision across US stakeholders leveraging innovative data, digital, and technology solutions to support the Rare Disease Business Unit. Guided by modern, cloud-based technologies and applying best practices, they will closely collaborate with the DD&T and the RDBU I&A teams to deliver and maintain effective solutions and help drive adoption.
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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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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.
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The candidate has responsibility, under the supervision of the Epi Director, who is the Program Lead for various Rare Disease to lead one of these indications. The candidate will be charged with understanding the observational data needs and opportunities to enhance drug development with the mRNA platform, and build expertise as needed within the Rare Disease portfolio.
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Principal Scientist/ Associate Director, Preclinical Pharmacology Triveni Bio is breaking new ground on the identification of novel disease targets – working at the convergence of human genetics, best-in-class antibody design, and precision medicine.
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Additional areas of interest could include computational methods for genome assembly, population genetics, functional genomics, or structural variant interpretation in rare disease. The team member will focus on leading research projects related to analyses of long-read sequencing and structural variation in population studies such as the All of Us Research Program and the genome aggregation database (gnomAD), as well as rare disease programs including the NHGRI Mendelian genomics program (GREGoR) and the NICHD Fetal Genomics Consortium.
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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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The Regional Medical Scientific Director (Medical Science Liaison) is a credentialed (i.e., PhD, PharmD, DNP, MD) Director Level Medical Affairs position and therapeutic and disease expert who engages in bidirectional exchange of scientific or medical information with Scientific Leaders (SLs.
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rare disease jobs Title: director Company: Moderna Therapeutics in Cambridge, MA
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