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dc.contributor.advisorKristala L. Jones Prather and Roy E. Welsch.en_US
dc.contributor.authorRodríguez Pinhao Miessner, Diegoen_US
dc.contributor.otherLeaders for Global Operations Program.en_US
dc.date.accessioned2016-09-13T19:13:26Z
dc.date.available2016-09-13T19:13:26Z
dc.date.copyright2016en_US
dc.date.issued2016en_US
dc.identifier.urihttp://hdl.handle.net/1721.1/104215
dc.descriptionThesis: S.M., Massachusetts Institute of Technology, Department of Chemical Engineering, 2016. In conjunction with the Leaders for Global Operations Program at MIT.en_US
dc.descriptionThesis: M.B.A., Massachusetts Institute of Technology, Sloan School of Management, 2016. In conjunction with the Leaders for Global Operations Program at MIT.en_US
dc.descriptionCataloged from PDF version of thesis.en_US
dc.descriptionIncludes bibliographical references (pages 51-56).en_US
dc.description.abstractGene therapy is a promising modality for the potential treatment of rare Mendelian diseases. To date a number of high profile proof-of-concept studies within the industry have demonstrated the significant disease-correcting promise of this therapeutic strategy. One of the major hurdles that remains for the commercialization of gene therapies is the lack of efficient manufacturing capabilities for the production of clinical-grade drug substance/drug product. The primary goals for this project were to decrease the biological contamination and cross-contamination risk associated with the biologic manufacturing process for viral gene therapy vectors and to adjust the process in order to optimize commercial profit. The project also included documenting the different existing processes for AAV production and developing a competitive analysis using information from ongoing clinical trials in the industry pipeline. The following process design steps were followed in order to fulfill the project objectives: (1) Define product specifications, analytical needs and market size, (2) Select production platform/process, (3) Collect data and create process flow diagram, (4) Perform material and energy balances, (5) Calculate costs: equipment and consumables, (6) Model the process in a spreadsheet, (7) Carry out sensitivity analyses, (8) Assess cost-effectiveness and risk, and (9) Develop recommendations. Five different AAV production platforms were identified and an AAV gene therapy landscape was generated. Also, the current process that Pfizer is planning to use was documented and an initial market sizing was performed. Finally, all the data necessary to model the process was collected and the cost-effectiveness and biological contamination and cross-contamination risk assessment were completed. This project confirmed that the use of a scalable line of single-use high cell density bioreactors for the production of AAV is cost-effective. This implies that sufficient AAV quantities can be manufactured for preclinical and clinical trials, using the process developed by Pfizer.en_US
dc.description.statementofresponsibilityby Diego Rodríguez Pinhao Miessner.en_US
dc.format.extent64 pagesen_US
dc.language.isoengen_US
dc.publisherMassachusetts Institute of Technologyen_US
dc.rightsM.I.T. theses are protected by copyright. They may be viewed from this source for any purpose, but reproduction or distribution in any format is prohibited without written permission. See provided URL for inquiries about permission.en_US
dc.rights.urihttp://dspace.mit.edu/handle/1721.1/7582en_US
dc.subjectChemical Engineering.en_US
dc.subjectSloan School of Management.en_US
dc.subjectLeaders for Global Operations Program.en_US
dc.titleProduction of AAV vectors for gene therapy : a cost-effectiveness and risk assessmenten_US
dc.title.alternativeProduction of Adeno-Associated Virus vectors for gene therapyen_US
dc.typeThesisen_US
dc.description.degreeS.M.en_US
dc.description.degreeM.B.A.en_US
dc.contributor.departmentLeaders for Global Operations Program at MITen_US
dc.contributor.departmentMassachusetts Institute of Technology. Department of Chemical Engineering
dc.contributor.departmentSloan School of Management
dc.identifier.oclc958140532en_US


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