Zimura (avacincaptad pegol): C5 Inhibitor
[for GA Secondary to AMD / Autosomal Recessive Stargardt Disease]

Zimura (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, Zimura 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 Zimura as a potential therapy for both geographic atrophy secondary to age-related macular degeneration and Stargardt disease. Clinical trials studying Zimura for these therapeutic applications are underway.
IC-500 (HtrA1 Inhibitor)

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.
IC-100 (RHO-adRP gene Tx)

IC-100 is delivered by an adeno-associated virus (AAV) 5 vector, for the treatment of rhodopsin-mediated, autosomal dominant retinitis pigmentosa (RHO-adRP). IC-100 is designed to simultaneously knockdown the production of the patient’s toxic rhodopsin protein produced by the mutated gene and replace it with a healthy rhodopsin protein via a single vector. IC-100 is in preclinical development.
IC-200 (BEST 1-Related IRDs

IC-200 is delivered by an adeno-associated virus (AAV) 2 vector, for the treatment of BEST 1-Related IRDs. IC-200 is designed to deliver a functional copy of the BEST 1 gene to retinal pigment epithelial cells to produce bestrophin-1 protein and normalize homeostasis between the photoreceptors and retinal pigment epithelial cells. IC-200 is in preclinical development.
miniCEP290 (Leber’s Congential 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.
miniABCA4 (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.
miniUSH2A: USH2A-Related IRDs (Usher's 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.