Improving vision and creating the world’s largest retinal gene therapy company
At the University of Oxford, Professor Robert MacLaren led the first clinical trials of gene therapy for choroideremia and X-linked retinal pigmentosa, both of which are inherited diseases leading to blindness, following his development of novel gene therapy vectors. This was the first time gene therapy had successfully been able to target the eye's photoreceptor cells and reverse visual field loss in retinitis pigmentosa.
This work led to the formation of a spinout company, Nightstar, in 2014. Licensing of subsequent research at Oxford, extending the approach to other kinds of retinal pigmentosa, created the world's largest retinal gene therapy company and the most comprehensive international gene therapy programme for any genetic disease to date.
Nightstar was listed on NASDAQ in 2017, and in 2019, the company was acquired by the multinational biotechnology company Biogen for USD877,000,000, representing the third most valuable British biotech exit in the last two decades.