Retinopathy of prematurity (ROP) is a leading cause of childhood blindness worldwide with serious unmet clinical need. It affects annually some 250,000 infants in the USA alone. It is caused by disturbed retinal oxygenation leading to neovascularization and vision loss. Hypoxia-inducible factor (HIF) pathway orchestrates hundreds of genes to coordinate processes, such as angiogenesis. This is a preclinical translational development program of HIA-01, an adeno-associated viral vector (AAV) delivering a single domain antibody, that specifically inhibits HIF-P4H-2. This leads to HIF stabilization, support of proper retinal vascularization and prevention of ROP. It is categorized as an advanced therapy medicinal product gene therapy. Unlike the current symptomatic treatment options, HIA-01 treats the disease so it does not develop. HIA-01 is expected to be curative with one intravitreal administration.

2024

2025