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:

Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry

Gene Therapy volume 24, pages 590–592 (2017)Cite this article

Subjects

Abstract

For the last 20 years, academic research has been the major, and often only, driving force behind the spectacular development of gene transfer technology for the therapy of rare genetic diseases. Investors and industry became eventually interested in gene and cell therapy, due to the success of a series of pioneering clinical trials that proved efficacy and safety of last-generation technology, and to favorable orphan drug legislation in both Europe and the United States. Developing this forms of therapy is however complex and requires skills and knowledge not necessary available to the industry, which is better placed to develop processes and products and put them on the market. Cooperation between academia and industry is an opportunity to de-risk innovative approaches and ensure a faster and more economical development of therapies for diseases with high unmet medical needs and low-profit expectations.

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

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

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

Similar content being viewed by others

References

  1. Abbot A . Gene therapy. Italians first to use stem cells. Nature 1992; 356: 465.

    Article  Google Scholar 

  2. Bordignon C, Notarangelo LD, Nobili N, Ferrari G, Casorati G, Panina P et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients. Science 1995; 270: 470–475.

    Article  CAS  Google Scholar 

  3. Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 1995; 270: 475–480.

    Article  CAS  Google Scholar 

  4. GSK Press Release Available at: http://www.gsk.com/en-gb/media/press-releases/2016/strimvelistm-receives-european-marketing-authorisation-to-treat-very-rare-disease-ada-scid/> 2016.

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

    Article  CAS  Google Scholar 

  6. Aiuti A, Vai S, Mortellaro A, Casorati G, Ficara F, Andolfi G et al. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Nat Med 2002; 8: 423–425.

    Article  CAS  Google Scholar 

  7. Aiuti A, Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A et al. Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J Clin Invest 2007; 117: 2233–2240.

    Article  CAS  Google Scholar 

  8. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009; 360: 447–458.

    Article  CAS  Google Scholar 

  9. Cavazza A, Moiani A, Mavilio F . Mechanisms of retroviral integration and mutagenesis. Hum Gene Ther 2013; 24: 119–131.

    Article  CAS  Google Scholar 

  10. Basner-Tschakarjan E, Mingozzi F . Cell-mediated immunity to AAV Vectors, evolving concepts and potential solutions. Front Immunol 2014; 5: 350.

    PubMed  PubMed Central  Google Scholar 

  11. Naldini L . Gene therapy returns to centre stage. Nature 2015; 526: 351–360.

    Article  CAS  Google Scholar 

  12. Meekings KN, Williams CS, Arrowsmith JE . Orphan drug development: an economically viable strategy for biopharma R&D. Drug Discov Today 2012; 17: 660–664.

    Article  Google Scholar 

  13. Lim WA, June CH . The principles of engineering immune cells to treat cancer. Cell 2017; 168: 724–740.

    Article  CAS  Google Scholar 

  14. Bryant LM, Christopher DM, Giles AR, Hinderer C, Rodriguez JL, Smith JB et al. Lessons learned from the clinical development and market authorization of Glybera. Hum Gene Ther Clin Dev 2013; 24: 55–64.

    Article  CAS  Google Scholar 

  15. Rama P, Matuska S, Paganoni G, Spinelli A, De Luca M, Pellegrini G . Limbal stem-cell therapy and long-term corneal regeneration. N Engl J Med 2010; 363: 147–155.

    Article  CAS  Google Scholar 

  16. GSK Press Release. Available at: https://us.gsk.com/en-us/media/press-releases/2010/gsk-fondazione-telethon-and-fondazione-san-raffaele-to-collaborate-on-gene-therapy-for-rare-diseases/> 2010.

  17. Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 2013; 341: 1233151.

    Article  Google Scholar 

  18. Hacein-Bey Abina S, Gaspar HB, Blondeau J, Caccavelli L, Charrier S, Buckland K et al. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. JAMA 2015; 313: 1550–1563.

    Article  Google Scholar 

  19. Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 2013; 341: 1233158.

    Article  Google Scholar 

  20. Audentes Press Release. Available at: http://investors.audentestx.com/phoenix.zhtml?c=254280&p=irol-newsArticle&ID=2123663> 2014.

  21. Childers MK, Joubert R, Poulard K, Moal C, Grange RW, Doering JA et al. Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. Sci Transl Med 2014; 6: 220ra10.

    Article  Google Scholar 

  22. Check Heyden E . Gene therapies pose million-dollar conundrum. Nature 2016; 534: 305–306.

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Genethon, Evry, France

    F Mavilio

  2. Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy

    F Mavilio

Authors
  1. F Mavilio
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to F Mavilio.

Ethics declarations

Competing interests

The author declares no conflict of interest.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Mavilio, F. Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry. Gene Ther 24, 590–592 (2017). https://doi.org/10.1038/gt.2017.36

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/gt.2017.36

This article is cited by

Search

Advanced search

Quick links