Fundraisings and IPOs

* On May 21, 2013,  uniQure, a human gene therapy company, has announced that with its consortium partners it is to receive a € 2.5 million Eurostars grant to develop an RNA interference (RNAi) gene therapy for Huntington’s disease. The consortium is a pan-European collaboration consisting of uniQure as the coordinator, Lausanne University Hospital, Switzerland, University Medical Center Göttingen, Germany, and Maria Curie-Sklodowska University, Poland.The program’s aim is to develop a gene therapy for the treatment of Huntington’s disease (HD), a rare and devastating neurodegenerative disease caused by mutations in the Huntingtin (Htt) gene. The program will start on June 1, 2013 and run for three years.

The consortium’s goals are to develop a regulated gene expression system for glial cell derived neurotrophic factor (GDNF) to improve the maintenance and survival of neurons as a HD gene therapy, and to develop regulated expression of artificial miRNA to conditionally silence the Htt gene. The main outcome of the program is a robust pre-clinical assessment of the first regulated gene therapy vector suitable for optimized treatment of HD patients. The ability to regulate gene expression would additionally represent an exciting innovation in the field of gene therapy, creating new opportunities to tackle challenging diseases where gene expression is only required at certain times. The consortium expects that the program’s results should lead to clinical safety trials within two years after the conclusion of the project.