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

LONDON--(BUSINESS WIRE) -- NightstaRx Ltd (“Nightstar”), a biopharmaceutical company specialising in developing gene therapies for inherited retinal dystrophies, today announces that the Company has commenced enrolling and dosing subjects in a Phase I/II clinical trial to treat patients with X-Linked Retinitis Pigmentosa (XLRP). This is the first clinical trial of its type in XLRP worldwide, and has been progressed from licensing to clinical trial in a period of just 18 months.

The gene therapy approach being used employs best in class innovation, using a viral vector known as adeno-associated virus (AAV) to deliver a codon-optimised copy of the retinitis pigmentosa GTPase regulator (RPGR) gene into cells of the eye, a first for clinical testing.

XLRP is a rare orphan disease resulting from a congenital genetic defect and one of the most common causes of blindness in young people. Progressive degeneration occurs due to loss of rod and cone photoreceptors. There are currently no treatment options.

Dr Aniz Girach, Chief Medical Officer of Nightstar commented “The current trial is a multicenter open-label, dose-escalation study designed to enroll at least 24 male patients at Ophthalmology centres of excellence, such as Oxford and Manchester. Each patient will receive a single subretinal injection of AAV-XLRPGR gene therapy. The primary goal of the study is to assess safety and tolerability of AAV- XLRPGR over a 12-month period.”

Robert MacLaren, Professor of Ophthalmology at the University of Oxford and principal investigator, commented: “X-linked retinitis pigmentosa is a devastating disease of early onset which leads to blindness in males. Many individuals are legally blind in their teens, and there is currently no treatment available. Based on previous findings in preclinical in vivo disease models, which have shown significant rescue of photoreceptors, we believe this approach has great potential to restore or maintain sight in patients. The unique codon-optimisation strategy overcomes the inherent instability problems of RPGR that confounded earlier attempts at gene replacement.”

Nightstar’s CEO David Fellows commented, “We are thrilled to report the advancement into the clinic of our second lead retinal dystrophy programme. If successful, this gene therapy has the potential of transforming the lives of many patients”

Notes for Editors:

About X-Linked Retinitis Pigmentosa

X-linked retinitis pigmentosa (XLRP) is an inherited X-linked recessive disease that inevitably leads to blindness in adult men, most often by the end of their fourth decade. XLRP constitutes a group of hereditary diseases that affect the retina’s ability to respond to light, specifically caused by abnormalities of the photoreceptors in the back of the eye. In more than two-thirds of cases, the disease is caused by a mutation to the retinitis pigmentosa GTPase regulator (RPGR) gene. Common early symptoms include difficulty seeing at night in young males and a progressive loss in the visual field and acuity. No effective treatment currently exists. For patients interested in participating in Nightstar’s clinical trial, please follow this link:

About Nightstar

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 is focused on building the world’s leading gene therapy company for the treatment of inherited retinal diseases. 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. The Company’s lead investors are Syncona Ltd, a publicly listed healthcare investment company taking an active role in identifying, developing, and funding technologies with the potential to significantly impact the healthcare market of the future, and NEA, a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors, and geographies.



David Fellows: +44 (0) 20 7611 2048

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