[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"detail-sidebar-cat-1-en-105":3,"doc-seo-170265-105":53,"doc-detail-170265-en":126},{"code":4,"msg":5,"data":6},0,"success",[7,14,19,24,29,34,39,44,49],{"id":8,"doc_module":9,"doc_module_name":10,"category_name":11,"show_sort_weight":12,"slug":13},11,1,"Template","Presentations",90,"presentations",{"id":15,"doc_module":9,"doc_module_name":10,"category_name":16,"show_sort_weight":17,"slug":18},12,"Resumes",80,"resumes",{"id":20,"doc_module":9,"doc_module_name":10,"category_name":21,"show_sort_weight":22,"slug":23},14,"Invoices",70,"invoices",{"id":25,"doc_module":9,"doc_module_name":10,"category_name":26,"show_sort_weight":27,"slug":28},15,"Posters",60,"posters",{"id":30,"doc_module":9,"doc_module_name":10,"category_name":31,"show_sort_weight":32,"slug":33},16,"Social Media",50,"social-media",{"id":35,"doc_module":9,"doc_module_name":10,"category_name":36,"show_sort_weight":37,"slug":38},17,"Forms",40,"forms",{"id":40,"doc_module":9,"doc_module_name":10,"category_name":41,"show_sort_weight":42,"slug":43},18,"Letters",30,"letters",{"id":45,"doc_module":9,"doc_module_name":10,"category_name":46,"show_sort_weight":47,"slug":48},21,"Paper Templates",5,"papers-templates",{"id":50,"doc_module":9,"doc_module_name":10,"category_name":51,"show_sort_weight":4,"slug":52},158,"General","general-158",{"code":4,"msg":54,"data":55},"ok",{"site_id":56,"language":57,"slug":58,"title":59,"keywords":60,"description":61,"schema_data":62,"social_meta":119,"head_meta":121,"extra_data":123,"updated_unix":125},105,"en","target-product-profile-tpp-template-gene-therapy","Target Product Profile (TPP) Template - Gene Therapy","","Target Product Profile (TPP) Template for the Platform Vector-Gene Therapy (PaVe-GT) program provides minimum acceptable and ideal attributes of an investigational AAV gene therapy drug product. It supports standardized planning across AAV gene therapies for rare diseases and enables public sharing of development artifacts. 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Furthermore, NIH disclaims any liability and provides no indemnification. A full list of terms and conditions for the use of PaVe-GT resources is available at \u0013 HYPERLINK \"https://pave-gt.ncats.nih.gov/terms-conditions-and-disclaimers/\" \\h \u0014pave-gt.ncats.nih.gov/ terms-conditions-and-disclaimers/\u0015.\nThe following template has been generated based on the NCATS Platform Vector Gene Therapy (PaVe-GT) team’s preparation of TPPs for their program. PaVe-GT is a pilot project that tests whether the efficiency of gene therapy trial startup can be improved through the standardization of processes across adeno-associated virus (AAV) gene therapies for four rare diseases. An important goal of PaVe-GT is to share project results and lessons learned with the public in such a way that the information may be used by any party interested in improving efficiency in the development of an AAV gene therapy. Specifically, as our projects progress through the different phases of drug development, we intend to make information publicly available. This includes FDA meeting requests and packages, toxicology and biodistribution data, Investigational New Drug (IND) filings and corresponding communications with the FDA, and other study documents. Please visit and subscribe to the PaVe-GT website for project updates and resources at \u0013 HYPERLINK \"http://pave-gt.ncats.nih.gov\" \\h \u0014pave-gt.ncats.nih.gov\u0015.\nInvestigators may use this tool to develop their own TPP for guiding their development program. An accompanying white paper, \u0013 HYPERLINK \"https://www.liebertpub.com/doi/10.1089/hum.2024.230\" \u0014AAV Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the PaVe-GT Program\u0015, describes the components of the TPP and the value it provides in drug development was recently published in Human Gene Therapy. We encourage reading the white paper for more details.\nInstructions for using the template:\nThis template includes the minimum acceptable and ideal attributes of the target drug product. While the ideal profile may not be achievable during early phases of development, it is helpful to describe it and to align the planned studies accordingly.\nThe template includes italicized blue text within brackets (intended to serve as guidance) and standard text in regular font. The italicized blue text along with the brackets should be substituted with information pertinent to the investigational product. The text in regular font may be utilized without modification.\n[Insert name of gene therapy product] Target Product Profile (TPP)\nProduct name: [Insert name of vector] vector expressing a functional [insert name of transgene], under control of the [insert name of promoter] promoter ([insert name of gene therapy product])\nTherapeutic Modality: Gene Therapy","cbCaidgiyqaVogsv","https://ap.wps.com/l/cbCaidgiyqaVogsv","docx",29529,"English","# Instructions for using the template\n## Template purpose and attributes\n## Guidance text to be replaced\n## Target drug product profile section\n## Product name and modality","[{\"question\":\"What is the purpose of the Target Product Profile (TPP) template?\",\"answer\":\"It defines minimum acceptable and ideal attributes for a target drug product, helping investigators align planned studies and standardize early planning for AAV gene therapy development.\"},{\"question\":\"How should investigators customize the template for their own product?\",\"answer\":\"Replace the italicized blue guidance text inside brackets with product-specific information for the investigational gene therapy.\"},{\"question\":\"What information is intended to be made publicly available as PaVe-GT projects progress?\",\"answer\":\"Planned public disclosures include FDA meeting requests and packages, toxicology and biodistribution data, IND filings and related FDA communications, and other study documents.\"}]","Target Product Profile (TPP) Template - Gene Therapy | DOCX",1788269562]