Gene therapy project wins £12m funding

A subsidiary of the Wellcome Trust has invested £12m in the development and commercialisation of therapies for retinal dystrophies following successful trial results last month.

London-based Syncona Partners has committed the funding to newly formed NightstaRx, a spin-out from the University of Oxford and its research commercialisation company Isis Innovation.

It follows the first clinical trial of a gene therapy for choroideraemia by a research team led by Professor Robert MacLaren of the Nuffield Laboratory of Ophthalmology at the University of Oxford (News 24.01.14).

The gene therapy used a small modified virus, AAV.REP1, to deliver the correct version of the choroideraemia gene to cells in the retina.