• Studies presented at leading ophthalmology and gene therapy conferences demonstrate therapeutic potential in geographic atrophy and wet AMD, leading causes of legal blindness
• This single-administration gene therapy approach could replace frequent eye injections for geographic atrophy and wet AMD patients
• The lead candidate IKAR-001 (previously known as IKC159V) will proceed to a geographic atrophy clinical trial supported by studies showing preservation of cells critical for vision

Norwich, UK, May 28 2025 – Ikarovec, a leader in ophthalmology innovation with a sophisticated field-changing technology for the long-term relief of prevalent eye diseases, has highlighted the therapeutic potential of its approach in geographic atrophy and wet AMD, with presentations at major ophthalmic and gene therapy conferences. The new preclinical data discussed at ARVO¹ and ASGCT² show how the company’s flexible bicistronic gene therapies can generate multiple therapeutic proteins where they are needed in the eye, protecting retinal cells and preventing damaging blood vessel growth and scarring.

The lead candidate IKAR-001 will proceed to a geographic atrophy clinical trial supported by studies showing preservation of cells critical for vision. The preclinical studies presented on the investigational agent, which encodes the therapeutically relevant proteins pigment epithelium-derived factor (PEDF) and soluble CD46, demonstrated its potential to improve clinical outcomes in geographic atrophy, an advanced common form of AMD.  

Additional studies presented at the conferences showed how another of the company’s bicistronic gene therapies reduced damaging new blood vessel formation, vascular leakage and scarring in preclinical models of wet AMD. Encoding inhibitors of vascular endothelial growth factor (VEGF) and connective tissue growth factor (CTGF), two proteins involved in driving disease progression and scarring respectively, this dual-target approach could enhance outcomes in wet AMD sufferers.

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About Ikarovec

Ikarovec is a leader in ophthalmology innovation, with a sophisticated field-changing technology for the long-term relief of prevalent eye diseases. The company’s flexible proprietary bicistronic gene therapy generates multiple synergistic therapeutic proteins where they are needed in the eye, bringing a differentiated approach to treating a range of large and growing ophthalmic disorders.

Its lead candidate, IKAR-001 (previously known as IKC159V), will proceed to a geographic atrophy (GA) clinical trial supported by studies showing preservation of cells critical for vision. GA leads to significant vision loss, affecting 5 million people worldwide. Current treatment options (only approved in the US) require regular injections directly into the eye. Ikarovec’s novel bicistronic gene therapy leads to the expression of two therapeutically relevant proteins, targeting multiple causes of the disease with a single injection which is expected to be disease modifying. Follow-on programmes are targeting wet age-related macular degeneration (AMD) and other chronic poorly managed ophthalmic indications.

Ikarovec is directed by an international Board and management team with industry-leading ophthalmology and gene therapy expertise. This experience comes via Astellas, EyeBio, OXB (Oxford Biomedica), Spark Therapeutics and others. Ikarovec, which is based at Norwich Research Park, is backed by LifeArc Ventures, Parkwalk Advisors and UKI2S. More information at www.ikarovec.com and on LinkedIn.