The Promise of Gene Therapy - Summary - 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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Objective:

To explore the potential of gene therapy, specifically RGX-314 (sura-vec), in transforming long-term treatment for wet age-related macular degeneration (nAMD).

Approach:
  • Gene Therapy Overview: Discussion on the advancements in gene therapy delivered subretinally as a drug delivery platform.
  • Upcoming Data: Anticipation of phase 3 topline data for sura-vec from the ATMOSPHERE and ASCENT trials.
Key Findings:
  • RGX-314 has the potential to reduce treatment burden for patients with nAMD.
  • Gene therapy may allow a subset of patients to avoid further injections after a single treatment.
  • Real-world treatment outcomes for nAMD are often inferior to those seen in clinical trials.
Interpretation:

Gene therapy could represent a paradigm shift in the management of nAMD, improving long-term outcomes.

Limitations:
  • Current findings are based on preliminary data; phase 3 results are pending.
Conclusion:

The implementation of gene therapy in clinical practice could optimize treatment for patients with wet AMD in the coming years.

Original Source(s)

The Promise of Gene Therapy

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

  • July 15, 2026

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