Preclinical data highlights potential of Ikarovec’s bicistronic gene therapy approach in common causes of vision loss

• 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 ASGCT 2 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.

Dr Thomas Ciulla, President & CEO of Ikarovec, said, “Sufferers of devastating eye diseases such as geographic atrophy and wet AMD are in real need of effective disease modifying treatments. The new preclinical data we have presented shows the potential of Ikarovec’s proprietary bicistronic gene therapy approach to generate these improved outcomes for patients, producing multiple therapeutic proteins in the eye to protect cells and reduce damage.” “In addition, our approach aims to dramatically reduce treatment burden on patients, by replacing frequent, often monthly, eye injections with a single administration, potentially improving both compliance and long-term outcomes,” he added. “Encouraged by the evidence generated to date and supported by our recent fundraising, we are working to complete advanced preclinical studies with our lead candidate IKAR-001, ready for the milestone of a clinical trial.”