Optician: Gene therapy project wins £12m funding

12 February 2014

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.

Results from the first six months of the trial, published in The Lancet medical journal, showed improvements in vision in dim light.

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