X-Linked Retinal Pigmentosa - Healing Genes

X-Linked Retinal Pigmentosa

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Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

An Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

Phase 1/2


Doctors at research centers in NY, NC, OK, OR, and TX seek pediatric patients with X-linked Retinitis Pigmentosa caused by specific genetic mutations in the RGBR gene. The drug uses a disabled adenovirus shell, delivered only within the retina, to deliver a functional copy of the RPGR gene. The harmless virus can selectively pass the corrected gene into the cells of the retina to enable normal function.

Treatment will involve a single injection surgery to the retina at the back of the eye, then follow up continues for 3 years.


  • Be 6 years of age or older and male
  • Meet genetic screening requirements specific to the study aim
  • Have good general health based on a complete physical examination and hematology and clinical chemistry studies performed at a pre-treatment evaluation


  1. Screening before the treatment.
  2. A single injection of the genetic therapy to the retina at the back of the eye.
  3. Follow up for 3 years.

The study has 9 U.S. locations, 5 of which are recruiting:
Columbia College of Physicians and Surgeons in New York, NY, Map.
Duke Eye Center, Duke University Medical Center in Durham, NC. Map.
Cincinnati Eye Institute in Cincinnati, OH, Map.
Casey Eye Institute, Oregon Health and Sciences University in Portland, OR. Map.
Retina Foundation of the Southwest in Dallas, TX. Map.
Jill Dolgin, PharmD  |  6178435781  |  [email protected]
Applied Genetic Technologies Corp
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