About the science

Increasingly, evidence from studies and scientific publications is converging to support the strong linkage between chronic local inflammation, activation of the Complement System, a part of the immune system, and AMD pathogenesis:

1. A number of complement system proteins, complement activators, and complement regulatory proteins have been identified as molecular constituents of drusen, the hallmark extracellular deposits associated with early AMD. Accumulation of these proteins in drusen may act as a depot of continual complement system stimulation, triggering the local production of inflammatory mediators, and attracting leukocytes that further augment the local inflammatory state driving AMD pathology.

2. Family history of AMD is a strong indicator of disease risk with the risk among siblings of an affected individual being three- to six-fold higher than the risk in the general population confirming the genetic component of this disease. Recent genetic studies performed on large cohorts of individuals have identified a set of gene variants as explaining at least 50% of the risk. Many of these genes encode key components of the complement system.

As overactivation of the complement system has been associated with the development of AMD, therapies which can restore the equilibrium of the complement system may significantly slow progression of AMD and prevent vision loss.

Gene therapy as a sustained drug delivery modality has proven effective and safe in ophthalmic and non-ophthalmic indications over several years now. Consistent levels of proteins of the complement system can only be achieved with gene therapy.