Introduction

Gene therapy looks to be coming of age, with seven projects in Phase III clinical development and one project currently under regulatory review. A shift in development from pure gene replacement to using gene therapy as a therapeutic delivery tool has enhanced the technology's commercial potential over the last 10 years.

Scope of this research

• In-depth analysis of the current gene therapy clinical pipeline.
• Analysis of key target indications for gene therapies and key companies active in the filed.
• Key opinion leader review of technologies driving clinical gene therapy.
• Identification of drivers and inhibitors to the commercial success of gene therapy.

Research and analysis highlights

Most of the indications being targeted in the current gene therapy pipeline are multifactorial rather than monogenic in etiology. Reflecting this trend, cancer has become the leading therapy area for gene therapy development, constituting nearly half of the clinical projects under development.

The development of highly engineered recombinant viruses has increased the safety of gene therapy. As clinical evidence for the enhanced safety of these vectors gathers over the next few years, the perceived risk/reward profile for gene therapy will change in some disease areas.

With endless lucrative targets for companies developing safe and efficient delivery of genes, the commercial potential of gene therapy remains high.

Key reasons to purchase this research

• Quantify the gene therapy pipeline.
• Assess the changing vectors and target indications for gene therapy.
• Identify the leading companies involved in gene therapy development.