Dual-Pathway Gene Therapy Targets Multiple Drivers in Retinal Disease - Takeaways - MDSpire

Dual-Pathway Gene Therapy Targets Multiple Drivers in Retinal Disease

At ARVO 2026, Ikarovec presented 4 posters showcasing its bicistronic platform that combines neuroprotection and complement modulation.

  • By

  • Katie Binley, PhD

  • May 1, 2026

  • 3 min

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  • 1

    Ikarovec is developing dual-pathway gene therapies for retinal degenerative diseases using a bicistronic gene therapy platform.

  • 2

    The lead program, IKAR-001, targets geographic atrophy by co-expressing PEDF for neuroprotection and soluble CD46 for complement modulation.

  • 3

    Current single-pathway therapies for geographic atrophy show modest effects and inconsistent improvements in visual function.

  • 4

    Neuroprotection is critical for meaningful outcomes in geographic atrophy, as it addresses neurodegeneration directly.

  • 5

    Ikarovec's gene therapy aims to provide both anatomical and functional benefits, representing a next-generation treatment for retinal diseases.

Original Source(s)

Dual-Pathway Gene Therapy Targets Multiple Drivers in Retinal Disease

  • by Katie Binley, PhD

  • May 1, 2026

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