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. 2021 Nov;28(10-11):615-617.
doi: 10.1038/s41434-020-0143-y. Epub 2020 Apr 2.

Toward a new framework for the development of individualized therapies

Peter Marks  1 , Celia Witten  2
Affiliations

Toward a new framework for the development of individualized therapies

Peter Marks et al. Gene Ther. 2021 Nov.
No abstract available

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Conflict of interest statement

The authors declare that they have no conflict of interest.

Figures

Fig. 1
Fig. 1. The current capacity for manufacturing of adeno-associated viral (AAV) gene therapy vectors is best suited for medium size populations ranging from roughly >100 to 10,000 individuals.
Current constrains on the technology for production related to the existing expression systems for making AAV preclude the production of the necessary quantities of product that would be required to address larger patient populations. At the other end of the spectrum, the current costs associated with product development preclude commercial viability of products that would be used in a very small number of individuals each year.
Fig. 2
Fig. 2. The potential benefits of a public–private consortium approach to the manufacturing of individualize gene therapy products include a significant reduction in the time for development.
The gene therapy product that would be produced with high quality at a site with significant manufacturing experience. Through use of templates and reference to master regulatory filings, it is expected that the burden of regulatory submissions could be reduced, and that ultimately the time from conception of a treatment to assessment of its effect could be reduced by about half or more.
See this image and copyright information in PMC

References

    1. Prickett TD, Crystal JS, Cohen CJ, Shamalov K, Trebska-McGowan K, Bliskovsky VV, et al. Durable complete response from metastatic melanoma after transfer of autologous T cells recognizing 10 mutated tumor antigens. Cancer Immunol Res. 2016;4:669–78. doi: 10.1158/2326-6066.CIR-15-0215. - DOI - PMC - PubMed
    1. Kim J, Hu CA, Moufawad El Achkar C, Black LE, Douville J, Larson A, et al. Patient-customized oligonucleotide therapy for a rare genetic disease. N Engl J Med. 2019;381:1678–80. doi: 10.1056/NEJMe1911295. - DOI - PMC - PubMed
    1. Woodcock J, Marks P. Drug regulation in the era of individualized therapies. N Engl J Med. 2019;381:1678–80. doi: 10.1056/NEJMe1911295. - DOI - PubMed
    1. Hampson G, Towse A, Pearson SD, Dreitlein WB, Henshall C. Gene therapy: evidence, value and affordability in the US health care system. J Comp Eff Res. 2018;7:15–28. doi: 10.2217/cer-2017-0068. - DOI - PubMed
    1. King A. A CRISPR edit for heart disease. Nature. 2018;555:S23–5. doi: 10.1038/d41586-018-02482-4. - DOI - PubMed

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