Neovascular Age-related Macular Degeneration (nAMD) - Healing Genes

Neovascular Age-related Macular Degeneration (nAMD)

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RGX-314 Gene Therapy for Neovascular AMD Trial

A Phase I/IIa, Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With RGX-314 in Subjects With Neovascular AMD (nAMD)

Phase 1 / 2


Doctors in Boston, MA, seek patients with Neovascular Age-related Macular Degeneration (nAMD) to trial an investigatory gene therapy. Through the delivery system of a genetically modified virus, a new gene will be delivered to the sub-retina in order to produce the soluble form of CD59 (sCD59). This CD59 is designed and intended to protect retinal cells by inhibiting the formation of the membrane attack complex (MAC), a cause of nAMD. The gene therapy can potentially permanently alter cells to make sCD59 for the life of the patient.

Participants will receive a single sub-retinal injection of the engineered virus, to be preceded and followed by intravitreal injections of anti-vascular endothelial growth factor (anti-VEGF). Follow up continues for 12 months.


  • Be 50 years of age or older
  • Diagnosis of treatment naive Wet AMD with no evidence of subretinal fibrosis under the fovea
  • Have adequate pupillary dilation to permit ocular examination and testing


  1. Screening to confirm acceptance to the study.
  2. Intravitreal anti-VEGF injection.
  3. Subretinal injection of gene therapy to the study eye.
  4. Intravitreal anti-VEGF injection monthly as needed.
  5. Follow up will continue for 12 months.

The study locations are Ophthalmic Consultants of Boston in Boston, MA, Map, and Vitreo-Retina Associates in Worcester, MA, Map.
Contact: Adam Rogers, MD  |  8572412276  |  [email protected]
Hemera Biosciences
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