Research report Assessment of preclinical gene therapy studies worldwide

Research reports | 04.09.2015 | CGM 2015-03

Frequently, the success of preclinical gene therapy studies in animal models is reported in the scientific literature and in the media. A survey of the worldwide-performed preclinical gene therapy studies and the successes reached, will give insight in future clinical trials and in the direction in which gene therapy will evolve. With this knowledge, COGEM is better prepared for future permit applications.

Although a survey of clinical trials can be found in several databases, this is not available in case of preclinical in vivo studies. Therefore, COGEM commissioned a research project to get insight in the worldwide-performed preclinical gene therapy studies and the obtained successes.

Based on the performed research, it seems that a growing amount of gene therapy medicines will enter the European market in the coming years. Consequently, gene therapy will acquire a permanent position in medicine. It is hard to predict how the Dutch society will respond to this development. Furthermore, the field of gene therapy is evolving continuously and the newest techniques are implemented, i.e. the application of exosomes, genome editing and vector barcoding. The impact of these developments on the environmental risk assessment remains a point of attention, which is monitored by COGEM conscientiously.

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