Gene Therapy Market Moving Towards Commercialization - Product Revenues Analyzed in New Research Report

Nov 20, 2015, 09:15 ET from RnR Market Research

PUNE, India, November 20, 2015 /PRNewswire/ --

2015 market research "Gene Therapy: Moving toward Commercialization" outlines the progress of the gene therapy field with a special focus on clinical-stage programs that are aimed at commercialization, and the companies that are carrying out these programs.

Complete report on gene therapy market spread across 187 pages, profiling 6 leading as well as start-up companies, offering survey data from 88 researchers involved in gene therapy and supported with 10 tables and 4 figures is now available at is now available at .

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 terms of expected revenues for gene therapy products reaching the market in the near term, there is a great deal of uncertainty. However, at least some analysts project sales of Lenti-D of around $250 million and of LentiGlobin of around $4 billion. As for the CAR T-cell therapies, one analyst projects peak sales of around $1.7 billion for Kite's KTE-C19. Other CAR T-cell therapies may achieve comparable results, depending on competition and payer acceptance of the therapies and their prices. These projections suggest that near-term gene therapies may attain commercial success.

Of the eight therapies, six are ex vivo gene therapies, which suggests that the ex vivo strategy (exemplified by bluebird bio) is a potentially successful one for moving gene therapies toward registration and marketing in the near term. Three of these ex vivo gene therapies are CAR T-cell cancer therapies that target CD19.

All of the eight therapies are for rare diseases. However, several of the diseases addressed by these therapies are for some of the more common rare diseases, especially beta-thalassemia and sickle cell disease. Thus the concern that gene therapy will only be applicable to ultra-rare diseases such as lipoprotein lipase deficiency (LPLD) is likely to be unfounded. However the prospect for gene therapies for common diseases has not yet been realized.

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; and Market outlook for eight gene therapy products. Order a copy of Gene Therapy: Moving Toward Commercialization report @

Explore more reports on gene therapy market and other research titles by Insight Pharma at

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