Nightstar Raises $45 Million to Fund Development of Gene Therapies for Blinding Retinal Diseases

Gene Therapy Leader Advancing Pipeline of Programs for Inherited Retinal Diseases, Including Upcoming Phase 3 Trial for Choroideremia.


NightstaRx Limited (“Nightstar”), a late-stage biopharmaceutical company developing gene therapies for inherited retinal diseases, today announced the completion of a $45 million Series C financing transaction. New investors Wellington Management Company and Redmile Group joined existing investors Syncona and New Enterprise Associates (NEA) in the round.

“Nightstar combines industry-leading gene therapy, ophthalmology expertise and clinical experience to advance a deep pipeline of therapies for devastating inherited retinal diseases,” said David Fellows, chief executive officer of Nightstar. “We are pleased to welcome these investors in the financing, which will provide the capital for us to move our pipeline forward so that we may help patients with these disorders maintain their vision.”

The Series C proceeds will be used for the upcoming Phase 3 trial for Nightstar’s lead product candidate NSR-REP1 for choroideremia, an ongoing Phase 1/2 clinical trial of Nightstar’s product candidate NSR-RPGR for X-linked retinitis pigmentosa, and a planned Phase 1/2 clinical trial of a gene therapy product candidate for an inherited form of macular dystrophy.

“As an original investor in Nightstar, our goal from day one was to build a global gene therapy leader with the capability of developing multiple programs for inherited retinal diseases. We welcome Wellington Management and Redmile Group as investors and look forward to working with them and NEA to fulfill Nightstar’s potential,” said Chris Hollowood, chairman of the board of Nightstar and chief investment officer of Syncona, a FTSE250 listed healthcare investment company founded by the Wellcome Trust.

About Nightstar

Nightstar is a leading retinal gene therapy company focused on developing and commercializing novel, potentially curative one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness, and for which there are no other treatment options. Nightstar’s lead program, NSR-REP1, is being developed as a one-time treatment for patients with choroideremia, a rare, inherited cause of progressive blindness that affects approximately one in every 50,000 people. The results from a successful Phase 1/2 trial of NSR-REP1 were published in the Lancet in 2014 and in the New England Journal of Medicine in 2016, and the program is advancing toward the first pivotal Phase 3 registrational clinical trial for this condition in the U.S., Europe, and Canada. Nightstar’s second gene therapy program, NSR-RPGR, is being evaluated in the first Phase 1/2 gene therapy clinical trial to treat patients with X-linked retinitis pigmentosa.

In addition to these two programs, Nightstar is evaluating several other retinal gene therapy product candidates for entry into clinical trials. The company was founded in 2014 by ophthalmology researchers 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 product candidates. These forward-looking statements involve substantial risks and uncertainties, and are based on the current expectations and assumptions of the Company’s management. You should not place undue reliance on the Company’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 release, and the Company does not undertake any obligation to update or revise any forward-looking statements, except as may be required by law.


Alicia Davis, THRUST IR

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