Gene Therapy: Barriers to Translation.

Davie N, Hyde SC, Barker R, Gill DR

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The British Society of Gene Therapy Annual Conference, London, 2014

Background:

In the 24 years since the first gene therapy was administered to a human patient (Blaese, 1995), the gene therapy industry has advanced significantly, culminating in the approval of Glybera (UniQure) in 2012. Despite this, the clinical and commercial success originally envisioned for gene therapy is yet to be realised. This poster examines the barriers preventing successful translation in the context of historical events in the field.

Methods:

The literature was reviewed and a Gartner Hype Cycle constructed to illustrate technology development. Experts in the field were consulted to populate the curve and comparisons were made with the evolution of another field, therapeutic antibodies.

Results:

Analysis of the curve suggests that gene therapy has matured as a technology and is currently on the ‘slope of enlightenment’. Expert opinion alludes to several barriers to effective translation. These include: investors' perception of high risk; limitations in the target market (orphan designation vs. crowded market); a poor reputation due to high-profile, early failures; uncertainty concerning the intellectual property landscape, and complex regulatory processes.

Conclusions:

Gene therapy has progressed since 1990, transitioning from academic bench to patient bedside. However, although understanding of the basic science has improved, other issues hinder its useful application. These translational barriers cannot be overcome through lab-based research. The limiting factor is not lack of scientific understanding, but uncertainty on the part of non-academic stakeholders, including industry, regulators, and clinicians.

Further work will assess how these groups could be engaged to streamline the translational pathway for gene therapy.

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