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FDA Provides Positive Feedback on IKAR-001 Development Plan

Ikarovec s advancing IKAR-001 with regulatory guidance supporting dual-pathway gene therapy approach

Norwich, UK – [Date] – Ikarovec Limited today announced positive feedback from recent interactions with the U.S. Food and Drug Administration (FDA) regarding the development pathway for IKAR-001, the company’s first-in-class dual-pathway gene therapy for geographic atrophy (GA).

The FDA feedback validated Ikarovec’s clinical development strategy and provided helpful guidance on the regulatory pathway as the company advances toward Investigational New Drug (IND) application submission.

“The positive FDA feedback reinforces our confidence in IKAR-001’s differentiated approach,” said Thomas Ciulla, MD, MBA, President and CEO of Ikarovec. “Our dual-pathway strategy—combining neuroprotection with complement modulation—is designed to preserve vision, not just slow disease progression. This represents a significant advancement over current single-pathway therapies.”

About IKAR-001

IKAR-001 uniquely combines neuroprotection with complement modulation in a single bicistronic AAV8 gene therapy. This dual-pathway approach builds on the validated complement pathway demonstrated by recently approved GA therapies while adding neuroprotection to address functional vision benefit—what current approved therapies miss.

Preclinical studies demonstrate IKAR-001’s superiority versus complement-only approaches. The gene-agnostic platform targets 5.0M GA patients worldwide with potential extension to 1.5M retinitis pigmentosa (RP) patients.

Regulatory and Clinical Progress

In addition to the positive FDA feedback, Ikarovec is advancing its Australian Clinical Trial Notification (CTN) application for Phase I/IIa trial initiation. The company has also:

• Initiated GMP manufacturing with an experienced AAV8 CDMO partner
• Assembled a world-class Scientific Advisory Board spanning Harvard, Oxford, NIH, University of Melbourne, Cornell, and Tel Aviv University
• Received strong validation at the American Academy of Ophthalmology’s EyeCelerator showcase in October 2025

About Ikarovec

Ikarovec is pioneering dual-pathway gene therapies for retinal degenerative diseases. The company’s proprietary bicistronic platform enables the expression of two synergistic therapeutic proteins from a single gene therapy vector, addressing multiple disease pathways simultaneously.

Led by an experienced management team with proven exits including Luxturna (Spark Therapeutics, $4.8B to Roche), Izervay (Iveric Bio, $5.9B to Astellas), and EyeBio ($3.0B to Merck), Ikarovec is backed by LifeArc Ventures, Parkwalk Advisors, and UKI2S.

Beyond IKAR-001, the company is developing office-based gene therapies for wet AMD (anti-angiogenic + anti-fibrotic) and intermediate AMD (neuroprotection + complement modulation).

For more information, visit www.ikarovec.com or follow us on LinkedIn.