UK gene therapy company gets more money

NightstaRx Ltd, a one-year old gene therapy company, has received £5 million in new venture finance to expedite the development of a treatment for choroideremia, an inherited cause of progressive blindness. This brings total funding for the company to £17 million.

NightstaRx Ltd, a one-year old gene therapy company, has received £5 million in new venture finance to expedite the development of a treatment for choroideremia, an inherited cause of progressive blindness. This brings total funding for the company to £17 million.

Nightstar is a spin-out of the University of Oxford and based on technology developed by Robert MacLaren, a professor at the university’s Nuffield Laboratory of Ophthalmology. The treatment uses an adeno-associated viral vector encoding the Rab escort protein 1 (REP1) to deliver a correct version of the choroideremia (CHM) gene to cells in the retina of the eye. Choroideremia is caused by mutations in the CHM gene and a loss of the REP1 protein. It leads to a progressive loss of vision due to the degeneration of the choroid and retina of the eye. There is currently no treatment or cure for the disease.

The Nightstar therapy is currently being investigated in a Phase 1/2 trial of 12 patients with choroideremia. Results from the treatment of six patients, reported in The Lancet in January 2014, showed improvements in their vision in dim light.

The latest funding has been provided by Syncona LLP with support from Oxford Spin-out Equity Management. Syncona also provided the initial £12 million in funding at the company’s launch in January 2014.

Separately, Nightstar announced the appointment of David Fellows as chief executive officer. Mr Fellows joined the company in 2014 as a member of the board of directors. He is formerly vice president of Johnson & Johnson Inc’s vision care business.

NightstaRx announced the new funding on 26 January 2015.

Copyright 2015 Evernow Publishing Ltd