Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Gene Therapy: Moving Toward Commercialization, by Allan B. Haberman, Ph.D.
This report looks at how researchers have been working to overcome critical barriers to development of safe and efficacious gene therapy, from 1990 to 2015. It then focuses on clinical-stage gene therapy programs that are aimed at commercialization, and the companies that are carrying out these programs. A major theme of the 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 gene therapy development.
Topics covered in the report:
- Development of improved vectors (integrating and non-integrating vectors)
- Gene therapy for ophthalmological diseases
- Gene therapy for hemophilias and other rare diseases
- Gene therapy for more common diseases (e.g., Parkinson’s disease, osteoarthritis, and heart failure)
- Companies whose central technology platform involves ex vivo gene therapy
- Gene editing technology
- Outlook for gene therapy
- Outlook for eight gene therapy products expected to reach the market before 2020
The report also 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, Spark Therapeutics, GenSight, Dimension Therapeutics, Voyager Therapeutics, Oxford BioMedica, GeneQuine, bluebird, Juno Therapeutics, Kite Pharma, Editas, and others.
For more information on the report, or to order it, see the CHI Insight Pharma Reports website.
June 6, 2015
Gene Therapy: Moving Toward Commercialization
Alan Haberman holds a Ph.D. in biochemistry and molecular biology from Harvard University and has an extensive background in the pharmaceutical and biotechnology industry.
More on the author.
*Report is available for single user, multi-user, single-site, multi-site or for global use.