News Release

Nightstar Appoints Tuyen Ong as Chief Development Officer

August 1, 2017

Board Certified Ophthalmologist and Industry Veteran Adds Clinical Development and Product Approval Expertise to Experienced Team

BOSTON and LONDON – August 1, 2017 -- NightstaRx Limited (“Nightstar” or the “Company”), a clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, today announced the appointment of Tuyen Ong, M.D., MRCOphth, MBA, a board-certified ophthalmologist and industry veteran, as executive vice president and chief development officer. Dr. Ong will be responsible for leading Nightstar’s global development efforts and clinical strategy, and helping to advance its proprietary pipeline towards regulatory approval. He will join Nightstar’s executive management team and report to David Fellows, the chief executive officer of Nightstar, and will be based out of the company’s U.S. headquarters in Lexington, Mass.

“Dr. Tuyen brings 20 years of clinical and drug development experience from both large pharma and biotech, working in the fields of ophthalmology and rare diseases, and we are thrilled to have him join our world-class team,” said Mr. Fellows. “Tuyen joins Nightstar at a very exciting time for us. As we advance our lead product candidate, NSR-REP1, into a planned Phase 3 clinical trial for the treatment of choroideremia in the first half of 2018 and conduct the ongoing Phase 1/2 clinical trial of our second product candidate, NSR-RPGR, for the treatment of X-linked retinitis pigmentosa, expanding our development capabilities in the U.S. will be critical. Tuyen’s experience in clinical development will help us as we seek to develop and commercialize novel, one-time gene therapy treatments for patients suffering from these rare inherited retinal diseases that would otherwise progress to blindness.”

Dr. Ong joins Nightstar from PTC Therapeutics Inc. (“PTC”), where he most recently served as chief medical officer. During his time at PTC, Dr. Ong was responsible for advancing the company’s pipeline of product candidates for the treatment of rare and orphan diseases. Prior to PTC, Dr. Ong served as vice president of global clinical development and operations at Bausch and Lomb Inc. (subsequently acquired by Valeant Pharmaceuticals International, Inc.) and played a key role in the company’s filing of new drug and device applications. Prior to that, Dr. Ong worked at Pfizer Inc. as its global clinical lead across multiple therapeutic disease areas of high-unmet need, most recently in retinal diseases. Dr. Ong holds an M.D. from the University College London and an MBA from New York University Stern School of Business. He is a member of the Royal College of Ophthalmologists.

“I’m excited to be joining Nightstar, a company at the forefront of gene therapy, and to be an integral part of its mission to develop cutting-edge treatments for rare inherited retinal diseases,” commented Dr. Ong. “Ophthalmic diseases are, in many ways, ideal targets for gene therapy. It’s a privilege to be joining such a strong team at this important time for the company. I look forward to working with Nightstar to continue to build relationships with patient advocacy groups, as well as the academic and regulatory communities, to help bring life-changing therapies to people with inherited retinal diseases and to better understand the diseases we are targeting.”

About Nightstar

Nightstar is a leading clinical-stage gene therapy company focused on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness. Nightstar’s lead product candidate, NSR-REP1, is being developed as a treatment for patients with choroideremia, a rare, degenerative, genetic retinal disorder that has no current treatments and affects approximately one in every 50,000 people. Positive results from a Phase 1/2 trial of NSR-REP1 were published in The Lancet in 2014 and in The New England Journal of Medicine in 2016.

Nightstar plans to commence a Phase 3 registrational trial of NSR-REP1 for choroideremia at sites in the United States, Europe and Canada. Nightstar’s second product candidate, NSR-RPGR, is currently being evaluated in a Phase 1/2 clinical trial for the treatment of patients with X-linked retinitis pigmentosa, an inherited X-linked recessive retinal disease that affects approximately one in every 40,000 people.

In addition to these two programs, Nightstar is evaluating other retinal gene therapy product candidates. Nightstar was founded in 2013 based on research by ophthalmologists at the University of Oxford. For more information about Nightstar or its clinical trials, please visit

Forward-Looking Statements

This release includes forward-looking statements regarding Nightstar’s plans for the clinical development of its gene therapy product candidates. These forward-looking statements involve substantial risks and uncertainties and are based on the current expectations and assumptions of Nightstar’s management. You should not place undue reliance on Nightstar’s forward-looking statements. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by the forward-looking statements in this release, including results of preclinical studies and clinical trials, receipt of regulatory approvals and market conditions. The forward-looking statements are made as of the date of this press release, and Nightstar does not undertake any obligation to update or revise any forward-looking statements, except as may be required by law.



Alicia Davis, THRUST IR