Combining new therapies

Ikarovec’s novel bi-functional gene therapy

Ikarovec’s flexible bi-functional gene therapy generates two beneficial proteins precisely where they are needed in the eye, bringing a differentiated approach to treating a wide range of ophthalmic disorders.
Ikarovec’s flexible bi-functional gene therapy generates two beneficial proteins precisely where they are needed in the eye, bringing a differentiated approach to treating a wide range of ophthalmic disorders.
Ikarovec’s gene therapies utilize a unique linker technology to ensure efficient processing and secretion of two independently acting therapeutic proteins.
Our gene therapies are designed to be delivered directly and safely to the back of the eye where the retinal disease originates.
Transduces target retinal cells to provide a sustained, lasting treatment solution.
Simultaneously expresses two proteins for enhanced efficacy in treating complex ophthalmic disorders.
1
Ikarovec’s gene therapies utilize a unique linker technology to ensure efficient processing and secretion of two independently acting therapeutic proteins.
2
Our gene therapies are designed to be delivered directly and safely to the back of the eye where the retinal disease originates.
3
Transduces target retinal cells to provide a sustained, lasting treatment solution.
4
Simultaneously expresses two proteins for enhanced efficacy in treating complex ophthalmic disorders.
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A unique approach delivering unparalleled benefits

Bi-functional Therapy

Expression of two therapeutic proteins from a single vector increases effectiveness

Once-only Treatment

To avoid the burden of regular injections associated with current therapies

Targeted Delivery

Vector delivery to the site of the disease concentrates therapeutic expression where it is most needed

Long-term Expression

This stable, long-term therapeutic approach reduces the reliance on complicated treatment plans

Targeting Age-Related Macular Degeneration

Dry-AMD and Geographic Atrophy

Disease Description
Dry Age-Related Macular Degeneration (Dry-AMD) is a progressive eye condition caused by the build-up of ‘drusen’ – deposits that accumulate beneath the retina. Over time, these deposits disrupt retinal pigment epithelial (RPE) cells, leading to the gradual breakdown of the retina. As the disease advances, areas of dead and dying (atrophic) retinal cells form patches, creating a pattern that resembles a map. This late stage of dry-AMD is known as Geographic Atrophy (GA), which can result in irreversible vision loss.
Geographic Atrophy
Our Solution
Ikarovec is developing an innovative bi-functional gene therapy to treat GA. Designed for targeted delivery to the back of the eye, this therapy transduces key retinal cells, enabling them to produce and secrete two clinically relevant proteins – Pigment Epithelium-Derived Factor (PEDF) and a soluble form of complement cofactor (sCD46). These proteins work synergistically to protect retinal cells, reduce inflammation, and limit abnormal blood vessel growth, offering a promising approach to slowing disease progression and preserving vision.
PEDF
Shown to reduce apoptosis, proinflammatory cytokine expression, and lipofusin accumulation.
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sCD46
Serves as a cofactor for complement factor I (CFI) to inhibit both the alternative and classical complement pathways

Wet-AMD

Disease Description
Wet Age-Related Macular Degeneration (Wet-AMD) is a more severe form of AMD that involves abnormal blood vessel growth beneath the retina, causing leakage and scarring. This can lead to rapid and debilitating vision loss and requires prompt treatment to manage symptoms and prevent further damage.
Wet-AMD
Our Solution
Ikarovec has developed a bi-functional gene therapy to treat wet-AMD. This therapy is specifically designed for targeted delivery to the back of the eye, where it transduces retinal cells and directs them to secrete two inhibitors – an anti-VEGF protein to reduce abnormal blood vessel growth, and an anti-connective tissue growth factor (anti-CTGF) protein to help prevent subretinal scar formation. Together, these proteins work to address the underlying causes of wet-AMD, offering a powerful new approach to preserving vision.
Targeting VEGF
To prevent pathological neovascularization and permeability.
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Targeting CTGF
A key driver of fibrosis to mitigate the formation of subretinal fibrosis.
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From Science to Sight

Dive into the groundbreaking research shaping our approach to treat macular degeneration.
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