Module 1
Administrations, N. A. (2023, 04 14). Code of Federal Regulations. Retrieved from https://www.ecfr.gov/current/title-21/chapter-I/subchapter-A/part-25?toc=1
CFR - Code of Federal Regulations Title 21. (2023, 03 28). Retrieved from U.S. Food & Drug Administration: https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=312.23
eCTD Resources. (2022, 12 13). Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/drugs/electronic-regulatory-submission-and-review/ectd-resources
Genetically Modified Organism (GMO) aspects for investigational medicinal products. (n.d.). Retrieved from European Commission: https://health.ec.europa.eu/medicinal-products/advanced-therapies/genetically-modified-organism-gmo-aspects-investigational-medicinal-products_en
BGTC Regulatory Playbook Version 1.0 Page 74
Investigational New Drug (IND) Application. (2022, 07 20). Retrieved from U.S. Food & Drug Administration – https://www.fda.gov/drugs/types-applications/investigational-new-drug-ind-application
Investigational New Drug Applications Prepared and Submitted by Sponsor-Investigators. (2015, May). Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/investigational-new-drug-applications-prepared-and-submitted-sponsor-investigatorsE6(R2) Good Clinical Practice: Integrated Addendum to ICH E6 (R1). (2018, March). Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/media/93884/download
Module 2
1. M4E(R2): The CTD – Efficacy. (2017, Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/m4er2-ctd-efficacy
2. M4S: The CTD — Safety. Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/m4s-ctd-safety
Module 3
Administration, U. F. (January 2020). Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs). Retrieved from https://www.fda.gov/media/113760/download
Beginner’s guide to the production of AAV vectors for gene therapy – Mentored by a CDMO: Guide to the production of AAV vectors for gene therapy - Biovian
21 CFR Part 211
21 CFR Part 600. Retrieved from https://www.ecfr.gov/current/title-21/part-600
Guidance for Industry CGMP for Phase 1 Investigational Drugs, July 2008. Retrieved from https://www.fda.gov/media/70975/download
Guidance for Industry Quality Systems Approach to Pharmaceutical CGMP Regulations, September 2006
Quality Systems Approach to Pharmaceutical Current Good Manufacturing Practice Regulations | FDA
U.S. Food and Drug Administration. Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs): Guidance for Industry. U.S. Department of Health and Human Services, Jan. 2020
U.S. Food and Drug Administration. Preclinical Assessment of Investigational Cellular and Gene Therapy Products: Guidance for Industry. U.S. Department of Health and Human Services, Nov. 2013, Preclinical Assessment of Investigational Cellular and Gene Therapy Products
Ricles, Laura. U.S. FDA’s Perspective on Delivery Devices Used to Administer Cell and Gene Therapy Products. CASSS Cell & Gene Therapy Products (CGTP): Manufacturing, Quality, and Regulatory Considerations, 27 June 2023, U.S. Food and Drug Administration, https://www.casss.org/docs/default-source/cgtp/2023-speaker-presentations/ricles-laura-fda-2023.pdf
Module 4
Administration, U. F. (2022, November 08). Study Data for Submission to CDER and CBER. Retrieved from https://www.fda.gov/industry/study-data-standards-resources/study-data-submission-cder-and-cber
Module 5
E6(R2) Good Clinical Practice: Integrated Addendum to ICH E6 (R1). (2018, March). Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/media/93884/download
Forms. (2023, 4 19). Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/about-fda/reports-manuals-forms/forms