Choroideremia - Healing Genes


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A Safety Study of Retinal Gene Therapy for Choroideremia (GEMINI)

An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia With Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

Phase 2


Doctors in Miami, FL, are recruiting adult male participants with choroideremia (CHM) to participate in an investigational trial of a gene therapy. This therapy is designed to improve choroideremia by delivering, through a safe viral transfer mechanism, a healthy copy of the gene that makes a protein missing in choroideremia.

Participants receive treatment in each eye at an interval to be determined by the investigators. The surgical procedure requires general anaesthesia and injection of the drug into a layer of the retina at the back of the eyes.


  • Be 18 years of age or older and male
  • Have a genetically confirmed diagnosis of CHM
  • Have active disease
  • Meet visual acuity criteria in both eyes
  • Not have a history of amblyopia or inflammatory eye disorder
  • Use contraception for a period of 3 months post treatment (both eyes)
  • Not have intraocular eye surgery in the 3 months before screening


  1. Prescreening to confirm eligibility of the patient to participate
  2. A surgery involving vitrectomy and injection of the investigational drug
  3. A second surgery for the second eye, at a period to be determined
  4. Further follow up will continue to the 2 years post treatment


The study site is in Miami, FL.

Contact: Nightstar Therapeutics | [email protected]

| +44 (0)207 062 2777 (international)



Nightstar Therapeutics

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