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Delivering advanced therapies: the big pharma approach

Abstract

After two decades of focused development and some recent clinical successes, cell and gene therapy (CGT) is emerging as a promising approach to personalized medicines. Genetically engineered cells as a medical modality are poised to stand alongside or in combination with small molecule and biopharmaceutical approaches to bring new therapies to patients globally. Big pharma can have a vital role in industrializing CGT by focusing on diseases with high unmet medical need and compelling genetic evidence. Pharma should invest in manufacturing and supply chain solutions that deliver reproducible, high-quality therapies at a commercially viable cost. Owing to the fast pace of innovation in this field proactive engagement with regulators is critical. It is also vital to understand the needs of patients all along the patient care pathway and to establish product pricing that is accepted by prescribers, payers and patients.

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References

  1. Sheridan C . Gene therapy finds its niche. Nat Biotechnol 2011; 29: 121–128.

    Article  CAS  PubMed  Google Scholar 

  2. Alliance for Regenerative Medicine 2016 Annual Report.

  3. Press Release Adaptimmune, June 2014.

  4. Bordignon C, Roncarolo MG, Aiuti A, Slavin S, Aker M, Ficara F et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296: 2410–2413.

    Article  PubMed  Google Scholar 

  5. Strimvelis Summary of Product Characteristics, June 2016.

  6. Van der Loo CMJ, Wright JF . Progress and challenges in viral vector manufacturing. Hum Mol Genet 2016; 25: 42–52.

    Article  Google Scholar 

  7. Skorska A, Müller P, Gaebel R, Große J, Lemcke H, Lux CA et al. GMP-conformant on-site manufacturing of a CD133 stem cell product for cardiovascular regeneration. Stem Cell Res Ther 2017; 8: 33–47.

    Article  PubMed  PubMed Central  Google Scholar 

  8. Mock U, Nickolay L, Philip B, Cheung G, Zhan H, Johnston I et al. Automated manufacturing of chimeric antigen receptor T cells for adoptive immunotherapy using CliniMACS prodigy. Cytotherapy 2016; 18: 1002–1011.

    Article  CAS  PubMed  Google Scholar 

  9. Priesner C, Aleksandrova K, Esser R, Mockel-Tenbrinck N, Leise J, Drechsel K et al. Automated enrichment, transduction and expansion of clinical-scale CD62L+ T cells for manufacturing of GTMPs. Hum Gene Ther 2016; 27: 860–869.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  10. Ortega V, Mohamed G, Ehman J, Zhu M, Mendiola C, Velagaleti G . Optimal strategy for obtaining routine chromosome analysis by using negative fractions of CD138 enriched plasma cells. Cancer Genet 2016; 209: 82–86.

    Article  CAS  PubMed  Google Scholar 

  11. Challice LB, Hollie JJ, Renier JB, Kevin JC . Toxicity and management in CAR T-cell therapy. Mol Ther Oncol 2016; 3: 415–419.

    Google Scholar 

  12. Bhatia SN, Ingber DE . Microfluidic organs-on-chips. Nat Biotechnol 2014; 32: 760–772.

    Article  CAS  PubMed  Google Scholar 

  13. Boheler KR, Gundry RL . Concise review: cell surface N-linked glycoproteins as potential stem cell markers and drug targets. Stem Cells Transl Med 2017; 6: 131–138.

    Article  PubMed  Google Scholar 

Download references

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Correspondence to J Tarnowski.

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The authors declare no conflict of interest.

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Special thanks to Amy Altshul and Damien O’Farrell.

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Tarnowski, J., Krishna, D., Jespers, L. et al. Delivering advanced therapies: the big pharma approach. Gene Ther 24, 593–598 (2017). https://doi.org/10.1038/gt.2017.65

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