Chapter 6: Gene Therapy and Viral Vectors: An Overview on Current Trends
56
5. Puri R K. Overview Office of Tissues and Advanced
Therapies and Division of Cellular and Gene Therapies
Research Program. OTAT-DCGT Overview. 8
May 2020. FDA website. https://www.fda.gov/
media/140940/download.
6. Kumar S R, et al. Clinical development of gene therapy:
results and lessons from recent successes. Mol Ther
Methods Clin Dev. 2016 3:16034.
7. Husain S R, et al. Gene therapy for cancer: regula-
tory considerations for approval. Cancer Gene Ther.
2015 22(12):554-63. 10.1038/cgt.2015.58.
8. Wang Z, et al. Detection of integration of plasmid
DNA into host genomic DNA following intra-
muscular injection and electroporation. Gene Ther.
2004 11(8):711-21.
9. FDA approves first-of-its kind targeted RNA-based
therapy to treat a rare disease. 10 August 2018. [FDA
News Release]. FDA website. https://www.fda.gov/
news-events/press-announcements/fda-approves-first-
its-kind-targeted-rna-based-therapy-treat-rare-disease.
10. Gardlik R, et al. Gene therapy for cancer: bacte-
ria-mediated anti-angiogenesis therapy. Gene Ther.
2011 18(5):425-31.
11. Baban C K, et al. Bacteria as vectors for gene therapy of
cancer. Bioeng Bugs. 2010 1(6):385-94.
12. Op cit 7.
13. Crystal RG. Adenovirus: the first effective in vivo gene
delivery vector. Hum Gene Ther. 2014 2. 5(1):3-11.
14. Wold W S, Toth K. Adenovirus vectors for gene ther-
apy, vaccination and cancer gene therapy. Curr Gene
Ther. 2013 13(6):421-33.
15. Bulcha J T, et al. Viral vector platforms within the
gene therapy landscape. Signal Transduct Target Ther.
2021 6(1):53.
16. Ibid.
17. Horowitz ED, et al. Biophysical and ultrastruc-
tural characterization of adeno-associated virus
capsid uncoating and genome release. J Virol.
2013 87(6):2994-3002.
18. Wang D, et al. Adeno-associated virus vector as a plat-
form for gene therapy delivery. Nat Rev Drug Discov.
2019 18(5):358-378.
19. Ibid.
20. Escors D, Breckpot K. Lentiviral vectors in gene
therapy: their current status and future potential. Arch
Immunol Ther Exp (Warsz). 2010 58(2):107-19.
21. Bulcha JT, et al. Viral vector platforms within the
gene therapy landscape. Signal Transduct Target Ther.
2021 6(1):53.
22. Guidance for Industry. Preclinical Assessment of
Investigational Cellular and Gene Therapy Products.
November 2013. FDA website. https://www.fda.gov/
media/87564/download.
23. Guidance for Industry. Expedited Programs for
Regenerative Medicine Therapies for Serious
Conditions. February 2019. FDA website. https://www.
fda.gov/media/120267/download.
24. Op cit 22.
25. Op cit 22.
26. Guidance for Industry. Long Term Follow-Up After
Administration of Human Gene Therapy Products.
January 2020. FDA website. https://www.fda.gov/
media/113768/download.
27. Ibid.
28. Op cit 26.
29. Chemistry, Manufacturing, and Control (CMC)
Information for Human Gene Therapy Investigational
New Drug Applications (INDs). Guidance for Industry.
January 2020. FDA website https://www.fda.gov/
media/113760/download.
30. Ibid.
31. Human Gene Therapy for Hemophilia. Guidance for
Industry. January 2020. FDA website. https://www.fda.
gov/media/113799/download.
32. Human Gene Therapy for Rare Diseases. Guidance for
Industry. January 2020. FDA website. https://www.fda.
gov/media/113807/download.
33. Human Gene Therapy for Retinal Disorders. Guidance
for Industry. January 2020. FDA website. https://www.
fda.gov/media/124641/download.
34. Guidance for Industry. Preparation of IDEs and INDs
for Products Intended to Repair or Replace Knee
Cartilage. December 2011. FDA website. https://www.
fda.gov/media/82562/download.
35. Testing of Retroviral Vector-Based Human Gene
Therapy Products for Replication Competent
Retrovirus During Product Manufacture and Patient
Follow-up. Guidance for Industry. January 2020. FDA
website. https://www.fda.gov/media/113790/download.
36. Recommendations for Microbial Vectors used for
Gene Therapy. Guidance for Industry. September
2016. FDA website. https://www.fda.gov/files/
vaccines,%20blood%20&%20biologics/published/
Recommendations-for-Microbial-Vectors-Used-for-
Gene-Therapy--Guidance-for-Industry.pdf.
37. Guidance for Industry. Potency Tests for Cellular and
Gene Therapy Products. January 2011. FDA website.
https://www.fda.gov/media/79856/download.
38. Multidisciplinary Gene Therapy. EMA website. https://
www.ema.europa.eu/en/human-regulatory/research-de-
velopment/scientific-guidelines/multidisciplinary/
multidisciplinary-gene-therapy.
39. Ensuring the Quality and Safety of Gene Therapy
Products. July 2019. PMDA website. https://www.
pmda.go.jp/files/000235607.pdf.
40. Expedited Programs for Regenerative Medicine
Therapies for Serious Conditions. Guidance for
Industry. February 2019. FDA website. https://www.
fda.gov/media/120267/download.
41. PRIME: priority medicines. EMA website. https://
www.ema.europa.eu/en/human-regulatory/research-de-
velopment/prime-priority-medicines.
42. Quality Guidelines. ICH website. https://www.ich.org/
page/quality-guidelines.
All URLs were accessed on 31 January 2022.
56
5. Puri R K. Overview Office of Tissues and Advanced
Therapies and Division of Cellular and Gene Therapies
Research Program. OTAT-DCGT Overview. 8
May 2020. FDA website. https://www.fda.gov/
media/140940/download.
6. Kumar S R, et al. Clinical development of gene therapy:
results and lessons from recent successes. Mol Ther
Methods Clin Dev. 2016 3:16034.
7. Husain S R, et al. Gene therapy for cancer: regula-
tory considerations for approval. Cancer Gene Ther.
2015 22(12):554-63. 10.1038/cgt.2015.58.
8. Wang Z, et al. Detection of integration of plasmid
DNA into host genomic DNA following intra-
muscular injection and electroporation. Gene Ther.
2004 11(8):711-21.
9. FDA approves first-of-its kind targeted RNA-based
therapy to treat a rare disease. 10 August 2018. [FDA
News Release]. FDA website. https://www.fda.gov/
news-events/press-announcements/fda-approves-first-
its-kind-targeted-rna-based-therapy-treat-rare-disease.
10. Gardlik R, et al. Gene therapy for cancer: bacte-
ria-mediated anti-angiogenesis therapy. Gene Ther.
2011 18(5):425-31.
11. Baban C K, et al. Bacteria as vectors for gene therapy of
cancer. Bioeng Bugs. 2010 1(6):385-94.
12. Op cit 7.
13. Crystal RG. Adenovirus: the first effective in vivo gene
delivery vector. Hum Gene Ther. 2014 2. 5(1):3-11.
14. Wold W S, Toth K. Adenovirus vectors for gene ther-
apy, vaccination and cancer gene therapy. Curr Gene
Ther. 2013 13(6):421-33.
15. Bulcha J T, et al. Viral vector platforms within the
gene therapy landscape. Signal Transduct Target Ther.
2021 6(1):53.
16. Ibid.
17. Horowitz ED, et al. Biophysical and ultrastruc-
tural characterization of adeno-associated virus
capsid uncoating and genome release. J Virol.
2013 87(6):2994-3002.
18. Wang D, et al. Adeno-associated virus vector as a plat-
form for gene therapy delivery. Nat Rev Drug Discov.
2019 18(5):358-378.
19. Ibid.
20. Escors D, Breckpot K. Lentiviral vectors in gene
therapy: their current status and future potential. Arch
Immunol Ther Exp (Warsz). 2010 58(2):107-19.
21. Bulcha JT, et al. Viral vector platforms within the
gene therapy landscape. Signal Transduct Target Ther.
2021 6(1):53.
22. Guidance for Industry. Preclinical Assessment of
Investigational Cellular and Gene Therapy Products.
November 2013. FDA website. https://www.fda.gov/
media/87564/download.
23. Guidance for Industry. Expedited Programs for
Regenerative Medicine Therapies for Serious
Conditions. February 2019. FDA website. https://www.
fda.gov/media/120267/download.
24. Op cit 22.
25. Op cit 22.
26. Guidance for Industry. Long Term Follow-Up After
Administration of Human Gene Therapy Products.
January 2020. FDA website. https://www.fda.gov/
media/113768/download.
27. Ibid.
28. Op cit 26.
29. Chemistry, Manufacturing, and Control (CMC)
Information for Human Gene Therapy Investigational
New Drug Applications (INDs). Guidance for Industry.
January 2020. FDA website https://www.fda.gov/
media/113760/download.
30. Ibid.
31. Human Gene Therapy for Hemophilia. Guidance for
Industry. January 2020. FDA website. https://www.fda.
gov/media/113799/download.
32. Human Gene Therapy for Rare Diseases. Guidance for
Industry. January 2020. FDA website. https://www.fda.
gov/media/113807/download.
33. Human Gene Therapy for Retinal Disorders. Guidance
for Industry. January 2020. FDA website. https://www.
fda.gov/media/124641/download.
34. Guidance for Industry. Preparation of IDEs and INDs
for Products Intended to Repair or Replace Knee
Cartilage. December 2011. FDA website. https://www.
fda.gov/media/82562/download.
35. Testing of Retroviral Vector-Based Human Gene
Therapy Products for Replication Competent
Retrovirus During Product Manufacture and Patient
Follow-up. Guidance for Industry. January 2020. FDA
website. https://www.fda.gov/media/113790/download.
36. Recommendations for Microbial Vectors used for
Gene Therapy. Guidance for Industry. September
2016. FDA website. https://www.fda.gov/files/
vaccines,%20blood%20&%20biologics/published/
Recommendations-for-Microbial-Vectors-Used-for-
Gene-Therapy--Guidance-for-Industry.pdf.
37. Guidance for Industry. Potency Tests for Cellular and
Gene Therapy Products. January 2011. FDA website.
https://www.fda.gov/media/79856/download.
38. Multidisciplinary Gene Therapy. EMA website. https://
www.ema.europa.eu/en/human-regulatory/research-de-
velopment/scientific-guidelines/multidisciplinary/
multidisciplinary-gene-therapy.
39. Ensuring the Quality and Safety of Gene Therapy
Products. July 2019. PMDA website. https://www.
pmda.go.jp/files/000235607.pdf.
40. Expedited Programs for Regenerative Medicine
Therapies for Serious Conditions. Guidance for
Industry. February 2019. FDA website. https://www.
fda.gov/media/120267/download.
41. PRIME: priority medicines. EMA website. https://
www.ema.europa.eu/en/human-regulatory/research-de-
velopment/prime-priority-medicines.
42. Quality Guidelines. ICH website. https://www.ich.org/
page/quality-guidelines.
All URLs were accessed on 31 January 2022.