NightstaRx, 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 1/2 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-optimized 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 centers 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: “XLRP 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-optimization 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 program. If successful, this gene therapy has the potential of transforming the lives of many patients”