Choroideremia AAV2-REP1 - Healing Genes

Choroideremia AAV2-REP1

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Efficacy and Safety of AAV2-REP1 for the Treatment of Choroideremia (STAR)

A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial Of Retinal Gene Therapy For Choroideremia Using An Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

Phase 3

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 in the eye chosen for the study
  • 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 subretinal injection of the investigational drug.
  3. Further follow up will continue for the year post treatment.

The study sites in CA, FL, MD, NY, OR, TX, and WI, are not yet posted. See contact info.
Nightstar Therapeutics | [email protected]  | +44 (0)207 062 2777 (international)
Nightstar Therapeutics
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