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Jul 24, 2025

FDA fast tracks one-time intravitreal gene therapy for geographic atrophy

By: Anthony DeFino Fact checked by: Christine Klimanskis, ELS

Key takeaways:

  • SAR446597 is designed to inhibit C1s and factor Bb in the complement pathway.
  • Fast-track designation expedites the development and review of treatments.

Perspective from Sunir J. Garg, MD, FACS, FASRS

The FDA granted fast track designation to SAR446597, an intravitreal gene therapy for the treatment of geographic atrophy due to age-related macular degeneration, according to a press release from Sanofi.

Sanofi is planning a phase 1/2 study to investigate safety, tolerability and efficacy of SAR446597 gene therapy.

The one-time injection is designed to target and inhibit two components of the complement pathway: C1s in the classical pathway and factor Bb in the alternative pathway.

“This dual-targeting approach potentially offers clinical advantages by providing sustained complement suppression within the retinal microenvironment while significantly reducing treatment burden through elimination of frequent intravitreal injections,” Sanofi said in the release.

Read more: https://www.healio.com/news/ophthalmology/20250717/fda-fast-tracks-onetime-intravitreal-gene-therapy-for-geographic-atrophy?utm_source=selligent&utm_medium=email&utm_campaign=20250720OSN&utm_content=20250720OSN

Source: Healio

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