An IV-Delivered Treatment for AMD and Neurodegenerative Ocular Diseases

Activated Protein C (APC) for the treatment of Age Related Macular Degeneration (AMD) delivered via IV, avoiding costly and painful ocular injections.

 

Neuroprotective therapy for ocular diseases is an urgent need in ophthalmology that remains challenging.

3K3A-activated protein C (APC), a genetically engineered variant of the endogenous protease APC, possesses neuroprotective, anti-inflammatory, anti-apoptotic, and vasculoprotective properties in the brain and nervous system.

The aim of this project is to develop this multitarget, multi-action molecule for the treatment of ocular diseases characterized by inflammation, retinal blood barrier dysfunction, retinal edema, and Choroidal Neovascularization (CNV)—the primary cause of vision impairment in patients with neovascular Age-Related Macular Degeneration (AMD).

3K3A-APC can be delivered via IV, avoiding costly and uncomfortable ocular injections, thus potentially revolutionizing the landscape of ocular care.

Inventors

Prof. Dov Weinberger, Rabin Medical Center
Prof. Tami Livnat, Rabin Medical Center
Yael Nisgav, Rabin Medical Center

Contact info

Sari Prutchi Sagiv PhD. Director of Pharma and Diagnostics

For further information please contact:

sari@mor-research.com

3K3A-APC can be delivered via IV, avoiding costly and uncomfortable ocular injections, thus potentially revolutionizing the landscape of ocular care.
Our laboratory results have demonstrated that 3K3A-APC functions as a pleiotropic cytoprotective molecule in the retina, exhibiting several key beneficial activities:
• Anti-inflammatory action
• Inflammasome inhibition
• VEGF reduction within the retina
• CNV growth suppression
• Retinal blood barrier stabilization
• Control of retinal leakage
• Counteraction of CNV-associated inflammation, which contributes to the ongoing neurodegenerative process in AMD.

Intravitreal administration is a commonly utilized method for delivering therapeutics to treat various retinal conditions. Our studies have confirmed its effectiveness for 3K3A-APC. However, considering patient comfort and the reduction of potential complications, we also examined the viability of intravenous administration. Preliminary experiments with this method have shown promising results, suggesting systemic treatment with 3K3A-APC could be a valuable therapeutic approach for patients. Importantly, intravenous administration has already been proven safe in clinical trials, positioning it as a short and cost-effective pathway toward human clinical trials for ocular applications of 3K3A-APC.

AMD accounts for 8.7% of all blindness worldwide, making it the most common cause of vision loss in developed countries. The disease significantly impacts daily activities like reading, driving, and recognizing faces. With an aging global population, the number of individuals affected by AMD is projected to rise from 196 million in 2020 to 288 million by 2040. The market for AMD treatments was valued at $7.3 billion in 2021 and is expected to grow at an annual rate of more than 11%.

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