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The company has a pipeline of novel and highly differentiated antibacterial and rare disease product candidates focused on patients with unmet need associated with multi-drug resistant (MDR) bacterial infections.
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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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This platform is fueling alliances with Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc. and Sangamo Therapeutics, Inc., as well as multiple programs in Voyager’s own pipeline.
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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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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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In this role, you will support programs in the discovery, preclinical, and/or clinical stages spanning multiple modalities including RNA editing, splicing and silencing, as well as several disease areas.
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Represent the voice ofindependent patient communities within Moderna, ensuring these perspectives shape the rare disease pipeline and related decisions. Develop and execute Patient Advisory Boards, Patient Leadership Councils and other forums in support of critical objectives pertaining to better understanding the patient true unmet need, gathering insights, supporting the amplification of the patient voice and ultimately, enabling Moderna to become embedded in the rare disease communities and ecosystem to better support patients and external stakeholders.
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Experience working in rare disease or gene therapy 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.
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Flare’s 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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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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Represent the voice of independent patient communities to internal stakeholders to ensure that patient perspectives are integrated into the rare disease pipeline decision-making processes.
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In addition to its clinical pipeline, Agios has multiple investigational therapies in preclinical development and an industry-leading research team with unmatched expertise in cellular metabolism and genetics.
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Prepare a business case including global revenue projections for a pre-clinical gene therapy program in a rare metabolic/liver targeted disease. One of the goals of this program is to feed a leadership pipeline of top talent for our Commercial Leadership Development Program (CLDP.
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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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The Director, Regulatory Affairs helps drive forward our regulatory strategy and operational activities related to Dyne’s muscle disease programs. 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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rare disease pipeline jobs in Lexington, MA
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