AMD therapies in Phase I

RTP-801i (Silence Therapeutics/Atugen): siRNA

RTP-801i is based on Atugen's proprietary siRNA technology that modifies the expression of the hypoxia-inducible gene RTP801, leading to the inhibition or reduction of CNV and vessel leakage that is involved in the progression of AMD.

Silence Therapeutics (formerly SR Pharma) recently launched a Phase I clinical trial with the agent.

POT-4 (Potentia Pharmaceuticals): C3 antagonist

POT-4 is a synthetic peptide that binds tightly to complement component C3, preventing its participation in the complement activation cascade. As C3 is a central component of the major complement activation pathways, its inhibition effectively shuts down all downstream complement activation that could otherwise lead to inflammation, tissue damage and upregulation of angiogenic factors such as VEGF.

The drug has recently entered into the clinical phase of development.

AdPEDF (GenVec): Adenovector

AdPEDF uses GenVec's proprietary adenovector, a DNA carrier, to deliver the human Pigment Epithelium-Derived Factor (PEDF) gene, resulting in the local production of AdPEDF in the treated eye. PEDF is normally produced in the eye and serves two important functions; regulating normal blood vessel growth and protecting the photoreceptors.

GenVec has completed the dose escalation portion of a Phase I, multicentre clinical trial in 28 patients with advanced wet AMD. Despite the advanced stage of disease in the patients studied, investigators observed positive changes in vision and retinal appearance at the higher dose cohorts in some patients. No dose limiting toxicities or drug-related severe adverse events were observed.