Therapeutics

Optherion's lead therapeutic for early-stage (‘dry’) AMD, and indicated renal diseases (DDD and aHUS) is the recombinant ‘protective’ human CFH protein.  The strategy is one of augmentation not unlike the approach taken in the treatment of diabetes with the use of insulin.  

In early stage AMD, by preventing the degradation of the macula, it is hoped that patients taking the recombinant ‘protective’ CFH protein will be protected from the more devastating late stage forms of the disease (i.e. wet AMD or geographic atrophy).  

In DDD and a subset of aHUS patients, it is hoped that the provision of the recombinant ‘protective’ CFH protein will protect against renal failure.

Optherion’s recombinant ‘protective’ human CFH is currently in pre-clinical development.

With respect to therapeutic pipeline, Optherion is pursuing a number of compounds which offer significant potential.  Specifically, the two dimensions of focus are:

• Development of therapeutics based on data showing that other genes are independently and causally associated with age-related macular degeneration.  Optherion has acquired a broad portfolio of intellectual property that supports development of therapeutics associated with these genes.
• Development of our lead CFH therapeutic in other diseases of the alternative complement cascade.  Recent, independent research from leading centers around the world has suggested that the CFH ‘risk’ form may be associated with a variety of other diseases, including coronary artery disease, myocardial infarction, stroke, Alzheimer Disease and others.  Future research will elucidate the extent and nature of this association.