X-Linked Retinitis Pigmentosa - Healing Genes

X-Linked Retinitis Pigmentosa

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A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa (XIRIUS)

A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

Phase 1 / 2 /3

Researchers in PA, OR, and FL are recruiting male X-Linked Retinitis Pigmentosa patients for an investigation of a gene therapy designed to impart a functional retinitis pigmentosa GTPase regulator gene (RPGR) to correct the disease. The treatment would require a single intravitreal (into the retina) injection of the drug, a genetically modified and harmless virus, at 1 of 6 dosage levels. The patient will be followed for ~ 2 years to assess for adverse safety events.


  • Be 10 years of age or older and male
  • Have documentation of a mutation in the RPGR gene
  • Not have participated in a gene therapy trial previously or a clinical trial with an investigational drug in the past 12 weeks


  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. Intravitreal injection of the gene therapy at one of several dose levels.
  3. Follow up for ~ 2 years to screen for adverse safety events

The study sites are not yet disclosed. Contact the study’s contact to qualify.
Nightstar Therapeutics  |  +44 207 611 2077 (international) |  [email protected]

Nightstar Therapeutics

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