Advertisement

PentaVision

Retinal Physician

Article

Dual-Pathway Gene Therapy for Geographic Atrophy

An approach currently being investigated would combine neuroprotection and complement modulation.

By: Thomas A. Ciulla, MD, MBA
Retinal Physician March 1, 2026 Vol 23, Issue March-April 2026 Page(s): 9-12

Clinical Scorecard: Dual-Pathway Gene Therapy for Geographic Atrophy

At a Glance

CategoryDetail
Condition
Key MechanismsNeuroprotection, complement modulation, oxidative stress, and inflammation
Target Population
Care Setting

Key Highlights

  • GA causes progressive and irreversible vision loss.
  • Current treatments have significant limitations, including high treatment burden and risk of neovascular conversion.
  • Dual-pathway gene therapy aims to address multiple pathological mechanisms with a single administration.
  • Key proteins involved in GA pathogenesis include CD46 and PEDF, which play crucial roles in neuroprotection and inflammation.
  • Approximately 9% annual risk of developing neovascular AMD in GA patients.

Guideline-Based Recommendations

Diagnosis

  • Diagnosis of GA should consider the presence of drusen, lipofuscin deposits, and visual acuity assessments.

Management

    Monitoring & Follow-up

      Risks

        Patient & Prescribing Data

        Gene therapy approaches are necessary for sustained therapeutic levels of PEDF, which may improve visual outcomes.

        Clinical Best Practices

        • Utilize dual-pathway approaches to address both neuroprotection and complement modulation.
        • Consider genetic risk factors affecting complement regulation and their implications in treatment planning.

        References