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NightstaRx Ltd.. (11/12/15). "Press Release: NightstaRx Announces the Start of a Miami Gene Therapy Study to Treat Choroideremia". London.

Region Region Miami, FL
  Country United States (USA)
Organisations Organisation NightstaRx Ltd.
  Today Nightstar Therapeutics plc (Nasdaq: NITE)
  Group Nightstar Therapeutics (Group)
  Organisation 2 University of Miami
Products Product gene therapy
  Product 2 clinical research
Persons Person Fellows, David (NightstaRx 201511 CEO)
  Person 2 Wilson, Julia (JW Communications 201209– before Vectura Group)

NightstaRx Ltd (“Nightstar”), the biopharmaceutical company specialising in gene therapy for retinal dystrophies, announces that the Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, has begun enrolling and dosing subjects in a Phase II clinical trial to treat Choroideremia (CHM). This gene therapy approach uses a viral vector known as adeno-associated virus (AAV) to deliver a wild-type copy of the Rab-escort protein 1 (REP-1) gene (AAV2-REP1) into cells of the eye.

The trial, which is sponsored by Bascom Palmer Eye Institute and donors interested in retinal research, is an open label study involving a total of 6 male patients, who will each receive a single dose of AAV2-REP1, supplied by Nightstar, via a sub retinal injection.

Choroideremia is an inherited X-linked recessive disease which inevitably causes blindness. It is caused by mutations to the CHM gene which encodes Rab-escort protein 1 and affects approximately 1 in 50,000 people. The first symptom of the condition is usually an impairment of night vision which often occurs in early childhood. This is followed by progressive narrowing of the field of vision, as well as a decrease in the ability to see details, culminating in blindness, most commonly in late adulthood. No effective treatment currently exists.

David Fellows, CEO of Nightstar said: “Gene therapy has huge potential as a treatment for many patients who are suffering from genetic causes of blindness. We have seen some very promising results from our choroideremia programme and have recently expanded our pipeline to include other inherited retinal dystrophy disorders as we continue to strive to restore or maintain sight in patients suffering from these devastating blinding diseases.”

Byron L. Lam, Professor at the Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine commented: “We are extremely excited and optimistic about the success of this gene therapy trial and are continuing to treat more patients with choroideremia with the hope that the knowledge gained will also help patients with other retinal diseases.”

– Ends –

Further information:

David Fellows
+44 (0) 207 611 2077

JW Communications
Julia Wilson
+44 (0) 781 8430877

Notes for Editors:

About NightstaRx Ltd

Nightstar is a private biopharmaceutical company 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. Gene therapy has the potential to be an effective treatment for choroideremia and a range of other retinal dystrophies. The Company’s lead investor, Syncona LLP, an independent subsidiary of the Wellcome Trust, is an evergreen investment company, taking an active role in identifying, supporting and developing technologies with the potential to significantly impact the healthcare market of the future.

About Bascom Palmer Eye Institute

Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, is ranked the nation’s best program in ophthalmology by U.S. News & World Report. As the largest ophthalmic care, research and educational facility in the southeastern United States, more than 250,000 patients with nearly every ophthalmic condition are treated each year and more than 12,000 surgeries are performed annually. Founded in 1962, Bascom Palmer has patient care facilities in Miami, Palm Beach Gardens, Naples, and Plantation, Florida. For additional information, please contact Marla Bercuson, executive director of business operations at (305) 326-6190, or visit the website at

About Choroideremia

Choroideremia is an inherited disorder that leads to progressive loss of vision due to degeneration of the choroid and retina which is caused by a lack of Rab Escort Protein-1 (REP-1) and occurs almost exclusively in males. The first symptoms occur in childhood, with night blindness being the most common first symptom. As the disease progresses, there is loss of peripheral vision or ‘tunnel vision’, and later a loss of central vision. Progression of the disease continues throughout the individual’s life, although both the rate and the degree of visual loss can vary, even within the same family. There is currently no treatment or cure for this disease.
For patients interested in participating in choroideremia trials please follow this link:

Record changed: 2017-04-02


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