Date Published
November 2015

Page Count 
187

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Gene Therapy: Moving Toward Commercialization

Cover-imageInsight Pharma Reports’ Gene Therapy: Moving Toward Commercialization”, outlines the progress of the gene therapy field since its inception in the 1970s, with a special focus on clinical-stage gene therapy programs that are aimed at commercialization, and the companies that are carrying out these programs. A major theme of this report is whether gene therapy can attain commercial success by the early-to-mid 2020s, which types of gene therapy programs have the greatest likelihood of success, and what hurdles might stand in the way of clinical and commercial success of leading gene therapy programs.

In accord with the focus of this report, we have been asking:

  • Whether gene therapy can attain commercial success by the early-to-mid 2020s,
  • Which types of gene therapy programs have the greatest likelihood of success,
  • What hurdles might stand in the way of clinical and commercial success of leading gene therapy programs.

In addition to chapters that focus on various areas of commercial gene therapy, this report includes:

  • An expert interview with Sam Wadsworth, Ph.D., the Chief Scientific Officer of Dimension Therapeutics and former Head of Gene Therapy R&D at Genzyme.
  • Survey data from 88 researchers involved in gene therapy
  • Companies profiled:  uniQure, Voyager Therapeutics, Oxford BioMedica, GeneQuine Therapeutics, Celladon Corporation, and bluebird

Topics covered:

  • Development of improved vectors (integrating and non-integrating vectors)
  • Gene therapy for ophthalmological diseases
  • Gene therapy for other rare diseases
  • Clinical-stage gene therapies for selected rare diseases other than hemophilias
  • Gene therapy for more common diseases
  • Companies whose central technology platform involves ex vivo gene therapy
  • CAR T-cell immunotherapy as an area of ex vivo gene therapy
  • Gene editing technology
  • Outlook for gene therapy
  • Market outlook for eight gene therapy products