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Develop methods to quantitatively measure the purity and heterogeneity of AAV gene therapy vectors, including empty capsid, aggregation, and capsid protein purity. advancing our platform of gene therapy programs through clinical development, starting with lead programs in hemophilia and metabolic disorders of the liver, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics.
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As a leading rare disease biopharmaceutical company, Ultragenyx uses multiple technology modalities including small molecule, biologic, mRNA and AAV gene therapy to make its products. Reporting to the Senior Manager of Materials Planning, Ultragenyx is seeking a highly motivated Supply Chain professional to help build the internal manufacturing and supply chain processes for our AAV gene therapy products.
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Asklepios BioPharmaceutical, Inc. (AskBio) is a leading, clinical-stage gene therapy company founded in 2001 based on the work of adeno-associated virus (AAV) gene therapy visionary and pioneer, Jude Samulski, PhD. Since that time, our commitment to developing life-saving medicines that can potentially cure genetic diseases has never wavered.
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Experience with AAV gene therapy products, sterile injectables, and particle characterization is preferred. Knowledge and experience using biochemical or biophysical methods to characterize AAV-based viral vectors is desired but not required.
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Evaluate and implement new analytical platforms, such as Mass Photometry, SEC-MALS, and HPLC, to characterize AAV particles. Investigate and evaluate cutting edge biophysical and analytical instrumentation for AAV, AAV-Ab characterization.
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Design and execute cell-based assays (AAV/lentiviral transduction, transfection, flow cytometry, immunostaining, and other plate reader assays). Develop and execute molecular biology workflows such as vector design and cloning, DNA/RNA extraction, RT-PCR, qPCR, and next-generation sequencing (Illumina and Nanopore.
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Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy. Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.
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We leverage our proprietary high-throughput AAV engineering platform to create viral vectors with cell-type and tissue level specificity, overcoming many of the biological limitations of early gene therapy efforts.
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Design and execute cell-based assays (AAV transduction, transfection, flow cytometry, luciferase assays, and immunofluorescence). Perform cell culture using Cellstacks for various cell types for AAV.
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Responsibilities entail the design and production of genetic tools for pioneering neuroscience research, involving DNA manipulation and optimization of viral technologies, notably Adeno-associated virus (AAV), with a strong focus on upholding rigorous quality control standards.
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Familiar with AAV gene therapy design and development. AAV vector design, titering, dosing. Oversee design and screening of new AAV constructs for discovery. Analyze and interpret data from in vitro and in vivo pharmacology &safety studies.
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As the software engineers closest to Anduril customers and end-users, Mission Software Engineers solve complex technical challenges of operational scenarios while owning the end-to-end delivery of winning capabilities for Fury: our high-performance, multi-mission group 5 autonomous air vehicle (AAV.
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Working within Fractyl’s Gene Therapy Research Team, the Sci II / Senior Scientist will be integral to the discovery and development of next generation AAV-based gene therapies for the treatment and cure of diabetes, obesity, and other metabolic diseases.
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Position SummaryThe Senior Director, AAV Cardiovascular Marketing Lead will lead, develop and implement strategic and operational marketing plans which will support the implementation and delivery of the core strategic imperatives for the products in the AAV franchise.
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Family Planning & Parenting Support: Fertility (eg, IVF, preservation), adoption, and gestational carrier coverage with additional benefits and resources to provide support from planning to parenting.
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