REF: IND Submission

Module 1

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

2. 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

3. eCTD Resources. (2022, 12 13). Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/drugs/electronic-regulatory-submission-and-review/ectd-resources

4. 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

5. 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

6. 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

1. 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

2. 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

Module 4

1. 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

1. 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

2. Forms. (2023, 4 19). Retrieved from U.S. Food & Drug Administration: https://www.fda.gov/about-fda/reports-manuals-forms/forms