The Promise of Gene Therapy - Scorecard - MDSpire

The Promise of Gene Therapy

With phase 3 topline data for sura-vec expected later this year, Arshad M. Khanani, MD, FASRS, reflects on how gene therapy could transform long-term treatment for nAMD.

  • By

  • Arshad M. Khanani, MD, MA, FASRS

  • July 15, 2026

  • 2 min

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Clinical Scorecard: The Promise of Gene Therapy

At a Glance

CategoryDetail
Conditionwet age-related macular degeneration (nAMD)
Key MechanismsGene therapy delivered subretinally as a drug delivery platform
Target PopulationPatients with wet age-related macular degeneration
Care SettingClinical practice for long-term treatment

Key Highlights

  • Phase 3 topline data for sura-vec expected later this year
  • RGX-314 (sura-vec) shows potential to reduce treatment burden
  • Gene therapy could transform long-term treatment for nAMD
  • First pivotal gene therapy program designed for nAMD
  • Real-world treatment outcomes are inferior to clinical trials

Guideline-Based Recommendations

Diagnosis

    Management

    • Consider gene therapy for reducing treatment burden in nAMD

    Monitoring & Follow-up

      Risks

        Patient & Prescribing Data

        Patients with chronic injections for nAMD

        Potential for large subset of patients to avoid further injections after one subretinal gene therapy injection

        Clinical Best Practices

        • Implement gene therapy in clinical practice to optimize long-term outcomes

        Related Resources & Content

          Original Source(s)

          The Promise of Gene Therapy

          • by Arshad M. Khanani, MD, MA, FASRS

          • July 15, 2026

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