NightstaRx Commences first Phase I/II Gene Therapy Clinical Trial for X-Linked Retinitis Pigmentosa

A team led by Prof Robert MacLaren at the University of Oxford have begun enroling participants in a Phase I/II gene therapy clinical trial for x-linked retinitis pigmentosa (RP) caused by the RPGR gene. The trial is being run by NightstaRx Ltd (Nightstar), a biopharmaceutical company specialising in developing gene therapies for inherited retinal dystrophies.

This is the first clinical trial of its type in x-linked retinitis pigmentosa (XLRP) worldwide. It has been progressed from licensing to clinical trial in a period of just 18 months.

The study is designed to enrol at least 24 male participants at ophthalmology centres of excellence, such as Oxford and Manchester. Each participant will receive a single subretinal injection of the gene therapy, called AAV-XLRPGR. The primary goal of the study is to assess safety and tolerability of AAV- XLRPGR over a 12-month period. On March 16, 2017, a 29 year old British man became the first person with X-linked retinitis pigmentosa to undergo gene therapy. The operation took place at the Oxford Eye Hospital.

The theory behind gene therapy is to treat the disease by repairing the abnormal gene. This is achieved by replacing the disease causing faulty gene with a “normal” copy into an individual’s cells. The most successful method to deliver the gene to the cells is by using a virus that has been genetically modified to carry human DNA. The virus being used is called an adeno-associated virus (AAV) vector.

This initial phase of the clinical trial is only recruiting participants from the UK. We will keep you up to date with any developments in the study.

About NightstaRx

Nightstar is a private biopharmaceutical company, spun-out from the University of Oxford in 2014, focused on the development of therapies for retinal dystrophies. The company’s lead programme is a retinal gene therapy for choroideremia, a rare inherited cause of blindness that affects around 1 in 50,000 people.


Target 5000

Target 5000 is a Fighting Blindness research project which aims to provide genetic testing for the estimated 5,000 people in Ireland who have a inherited retinal condition. The purpose of this project is to provide a precise diagnosis and more detailed information about the nature and inheritance pattern of the condition. This information will be to a national patient registry which will enable patients who are eligible for clinical trials to be identified. Click here for more information about Target 5000 and how you can get involved.

Counselling Services

Being diagnosed with a condition causing sight loss can be very difficult. The Fighting Blindness Insight Counselling Service provides a range of supports for people affected by sight loss and their families. Click here for more information about the Insight Counselling Service or contact Insight on 01 674 6496 or