Nightstar Therapeutics plc (Nasdaq: NITE)

https://www.nightstartx.com

At Nightstar, our mission is to maintain and restore sight in patients with inherited retinal diseases. We are a clinical-stage company focused on developing and commercializing a pipeline of novel and potentially curative, one-time retinal gene therapies for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness, and, for which, there are no currently approved treatments. Our lead retinal gene therapy product candidate, NSR-REP1, is being developed for the treatment of choroideremia (CHM), a rare, degenerative, X-linked genetic retinal disorder primarily affecting males that is caused by a mutation in the CHM gene. We have an ongoing Phase 3 registrational clinical trial, known as the STAR trial, of NSR-REP1 for CHM. We anticipate that STAR study will be fully enrolled by the first half of 2019 and expect the one-year follow-up results of the STAR trial to be available in 2020. We are also currently conducting a prospective, natural history study, known as the NIGHT study, across multiple clinical sites in the United States, Europe and Canada. NSR-REP1 has been granted orphan drug designation for the treatment of CHM from both the U.S. Food and Drug Administration and the European Medicines Agency. We are developing NSR-RPGR for the treatment of X-linked retinitis pigmentosa (XLRP), an inherited X-linked recessive retinal disease characterized by mutations in the RPGR gene, leading to a lack of protein transport and a loss of photoreceptors, the specialized cells in the eye that convert light into visual signals. NSR-RPGR is currently being evaluated in a dose-ranging Phase 1/2 clinical trial for the treatment of XLRP, known as the XIRIUS trial. We expect the initial data on safety and tolerability from the dose escalation cohorts of this trial to be available towards the end of 2018; this data will determine the dose for study in an expansion cohort. We have also initiated a prospective, natural history observational study, which we refer to as the XOLARIS study, to better understand the progression of untreated XLRP. We have received orphan drug designation for NSR-RPGR for the treatment of XLRP from the European Medicines Agency. We also have product candidates in preclinical development for a number of inherited retinal diseases for which there are no approved treatments such Stargardt disease and Best vitelliform macular dystrophy, or Best disease. We are evaluating other in-licensing opportunities to broaden our pipeline and drive future growth. *

	Start	2017-08-01 renamed date ca
	Group	Biogen (Group)
	Predecessor	NightstaRx Ltd.
	Industry	gene therapy
	Industry 2	ophthalmic
	Person	Fellows, David (Beacon Therapeutics 202306 CEO formerly NightstaRx 201511 CEO)
	Person 2	Sundaram, Senthil (Terns Pharmaceuticals 202101 CEO before NightstaRx + Intercept + Lehman Brothers/Barclays)

	Region	London, Greater London
	Country	United Kingdom (GB)
	Street	9–10 Midford Place
	City	W1T 5BJ London
	Tel	+44-20-7062-2777
		Address record changed: 2020-12-02

Basic data	Employees	n. a.

		�About Section� taken from web site of organisation on 28.09.

		Record changed: 2023-07-10