Therapeutic Areas

Transforming Treatment for Chronic Eye Diseases

Age-related macular degeneration (AMD) is a common eye condition that primarily affects people over the age of 50, resulting in the loss of central vision.
Loss of central vision not only makes everyday tasks like reading, driving, or recognizing faces challenging but also profoundly impacts independence and quality of life. Living with AMD can be emotionally and practically challenging, leaving many older adults facing mental health struggles and increasing the risk of falls and social isolation.

>50%

Over half of AMD cases affect both eyes

x2-3

some life-style choices, such as smoking, increase the risk of developing AMD

288 m

The projected number of people affected by AMD globally by 2040
Healthy
Geographic Atrophy
Wet AMD
Stages of Age-related Macular Degeneration (AMD)
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Therapeutic Program 1

Therapeutic Program 2

Dry-AMD

Dry-AMD develops when the cells of the macula become damaged due to lack of nutrients and there is a build-up of waste products from the biochemical processes responsible for vision called drusen. The loss of vision is gradual, occurring over many years

Geographic Atrophy

As dry-AMD progresses, the continued build-up of drusen causes areas of retinal cell death and degeneration known as atrophy. This advanced stage of AMD is called Geographic Atrophy (GA). GA is marked by the progressive loss of retinal pigment epithelium (RPE) and photoreceptors (PRs) leading to irreversible vision impairment. GA can affect one or both eyes, with the risk of developing it in the second eye being higher if the first eye is already affected. Current treatments for GA offer limited efficacy with continued vision loss.

By increasing the expression of Pigment Epithelium-Derived Factor (PEDF) and soluble complement cofactor (sCD46), our goal is to protect the retinal cells, reduce inflammation, and limit abnormal blood vessel growth, potentially slowing disease progression and limiting conversion to wet-AMD.

Wet-AMD

Wet-AMD occurs when abnormal blood vessels grow beneath the macula, leading to the disruption of retinal cells. Without treatment, vision loss can progress quickly. Anti-VEGF eye injections are effective for many patients, but require regular administration directly into the eye and may become less effective over time. Whilst many patients respond well to anti-VEGF therapy, a significant portion go on to develop sight limiting subretinal fibrosis.

By increasing the co-expression of a modified Anti-Vascular Endothelial Growth Factor (anti-VEGF) capture protein and an anti-fibrotic protein targeting Connective Tissue Growth Factor (CTGF), the aim is to reduce pathological neovascularization and prevent the formation of fibrotic scars that can lead to significant vision loss. 

 

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Learn About Our Therapeutic Pipeline

Discover how we’re addressing unmet needs in AMD through innovative gene therapy pipeline
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