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European Biopharmaceutical Review

Gene Therapy

In 2014, following a quarter of a century roller coaster trajectory, Ledley et al published very optimistic conclusions after their examination of life cycles of gene therapy, stating “Gene therapy technologies are now achieving the level of maturity that innovation research and biotechnology experience suggest may be requisite for efficient product development” (1). Looking at the following, it seems that reality meets those odds:

• The first marketing authorisations granted in the Western countries (Strimvelis® for the rare immunodeficiency syndrome adenosine-deaminase-severe combined immunodeficiency (ADA-SCID), Imlygic® [HSV1-GMCSF] for melanoma, Kymriah® for acute leukocytic leukemia, Yescarta® for non-Hodgkin lymphoma, Luxturna® for Leber congenital amaurosis)
• The steady maturation of the field since the 1980s (>40,000 papers, nearly 20,000 patents, >2,500 clinical trials including >100 late stage Phase 2/3 or Phase 3 trials)
• Billions in capital investments and initial public offerings (IPOs) in gene therapy companies, the activity being now driven by large pharmaceutical companies (with a peak reached recently with the $8.7 billion acquisition of Avexis by Novartis)

Gene therapy is defined as the introduction of genetic material into patients with an intended curative, life-extending, or life-enhancing therapeutic benefit. The applications rely on a myriad of possibilities, including the transient or permanent genetic modification of the administered product or ex vivo-engineered tissue to produce proteins for internal, local, or systemic use, helping to replace or correct faulty genes (in the case of rare diseases), providing stimuli to regenerate tissues (in the case of brain or cardiac disorders), killing tissues, or enhancing defence mechanisms (in the case of cancer or infectious diseases).

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Dr Serge Braun PharmD, PhD, is Scientific Director of AFM-Téléthon, President of Genosafe (a CRO company dedicated to quality control of biotherapeutic products) and Director Neuromuscular diseases at Généthon. He has 10 years of experience in the neuromuscular diseases field in the academic sector and 10 years in the biotech sector as Vice President of Research of Transgene SA. Serge was also co-founder of Neurofit, a CRO specialised in preclinical testings, and Vice President of Alsace BioValley, the non-profit making organisation for the development of a major biotech cluster in Europe. He also serves as advisor for Venture Capital companies and bioclusters.
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