Avacincaptad pegol
Autosomal recessive Stargardt disease


Avacincaptad pegol is designed to target and inhibit the cleavage of complement protein C5 and the formation of its downstream fragments, C5a and C5b. By inhibiting the formation of these fragments, avacincaptad pegol is believed to decrease or slow the chronic inflammation and cell death associated with the retinal aging process by decreasing the formation of membrane attack complex (MAC) and inflammasome activity. Thereby potentially avoiding or slowing the degeneration of retinal pigment epithelial cells. This potential mechanism is the rationale for avacincaptad pegol as a potential therapy for Stargardt disease. Clinical trials studying avacincaptad pegol for these therapeutic applications are underway.

IC-500: HtrA1 inhibitor
Geographic atrophy


HtrA1 is a member of the serine protease family of enzymes. Overexpression of HtrA1 has been implicated in the progression of macular degeneration.1 Iveric Bio is developing a specific inhibitor of HtrA1 which targets both intracellular and extracellular HtrA1. Based on this and other characteristics we believe that IC-500 has the potential to be best-in-class. IC-500 is in preclinical development.

mini-CEP290: LCA10
Leber congenital amaurosis type 10


Leber’s congenital amaurosis type 10 (LCA10) is the most common type of LCA and is caused by mutations in the CEP290 gene. The large size of the CEP290 gene exceeds the packaging capacity of standard AAV vectors. Similar to our other minigene programs, we are seeking to develop a smaller CEP290 construct that fits inside a well-defined AAV vector and transcribes a functional protein. Our miniCEP290 program is in the late research stage.

mini-ABCA4: STGD1
Autosomal recessive Stargardt disease


Autosomal Recessive Stargardt Disease (STGD1) is caused by mutations in the ABCA4 gene. The large size of the ABCA4 gene exceeds the packaging capacity of standard AAV vectors. Similar to our other minigene programs, we are seeking to develop a smaller ABCA4 construct that fits inside a well-defined AAV vector and transcribes a functional protein. Our miniABCA4 program is in the research stage.

mini-USH2A
Usher syndrome type 2A


Usher syndrome type 2A (USH2A) is a type of Usher syndrome caused by mutations in the USH2A gene, which encodes a protein, usherin, believed to be important in the development and maintenance of neurosensory cells in the retina and the inner ear. The large size of the USH2A gene exceeds the packaging capacity of standard AAV vectors. Similar to our other minigene programs. We are seeking to develop a smaller USH2A construct that fits inside a well-defined AAV vector and transcribes a functional protein. Our miniUSH2A program is currently in the research stage.