New trial starts to treat X-linked Retinitis Pigmentosa

20 March 17

written by:

Jo Harford

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Fight for Sight is delighted to note the announcement made today by the University of Oxford that researchers have started a new gene therapy clinical trial to treat X-linked retinitis pigmentosa (XLRP) with the first patient undergoing the therapy on 16 March 2017.  XLRP is currently untreatable and leads to progressive and irreversible sight loss.

The trial is being run by NightstaRx Ltd (NighstaRx), a spin out company from Oxford that is developing gene therapies for inherited retinal diseases and a team of researchers at the University of Oxford led by Professor Robert MacLaren. The main purpose of the trial is to assess the safety and tolerability of the gene therapy over a 12 month period and it is expected that at least 24 patients will be enrolled in the trial.

Fight for Sight has been a key funder of the gene therapy research in Oxford that has enabled this trial to commence. In particular, this trial would not have happened without the ground breaking research that has led to trials of gene therapy to treat choroideremia (a rare inherited disorder that also causes progressive sight loss) which were greatly facilitated by the Tommy Salisbury Choroideremia Fund at Fight for Sight.

Michele Acton, CEO of Fight for Sight, said: “This is the world’s first trial to test a treatment for retinitis pigmentosa caused by the RPGR gene which is essential for the functioning of the light sensitive cells at the back of the eye.  We are very proud that Fight for Sight’s support for research in this area has helped lead to the start of another clinical trial.  This could make a significant difference to the lives of so many people and we wish the team in Oxford continued success in the coming months”.

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