Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Review
  • Published:

Delivering advanced therapies: the big pharma approach

Gene Therapy volume 24, pages 593–598 (2017)Cite this article

Subjects

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.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on SpringerLink
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

Figure 1
Figure 2
Figure 3

Similar content being viewed by others

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

Author information

Authors and Affiliations

  1. GlaxoSmithKline Research and Development, SVP Cell and Gene Therapy Platform, King of Prussia, PA, USA

    J Tarnowski, D Krishna, L Jespers, A Ketkar, R Haddock, J Imrie & S Kili

Authors
  1. J Tarnowski
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. D Krishna
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. L Jespers
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. A Ketkar
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. R Haddock
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. J Imrie
    View author publications

    You can also search for this author in PubMed Google Scholar

  7. S Kili
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to J Tarnowski.

Ethics declarations

Competing interests

The authors declare no conflict of interest.

Additional information

Special thanks to Amy Altshul and Damien O’Farrell.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

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

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/gt.2017.65

This article is cited by

Search

Advanced search

Quick links