Aniridia-associated keratopathy (AAK) is a rare genetic eye condition caused by PAX6 haploinsufficiency, which leads to chronic inflammation, neovascularization and vision loss. Currently, there are limited therapeutic options for the treatment of AAK, which has been linked to limbal stem cell deficiency.
Hypoxia-inducible factors (HIFs) play a central role in how retinal cells respond to low oxygen, but their chronic activation has been linked to the development of age-related macular degeneration (AMD) and related retinal disorders.
Primary open-angle glaucoma (POAG) is among the leading causes of irreversible blindness. Early detection and treatment are crucial for vision preservation, and research to identify genes associated with POAG is ongoing.
Pyrotech (Beijing) Biotechnology Co. Ltd. has identified gasdermin-E (GSDME) inhibitors reported to be useful for the treatment of genetic diseases, cancer, viral infection, age-related macular degeneration, autoimmune diseases, neurological disorders, renal disorders and cancer therapy-associated disorders.
Cirrus Therapeutics Inc. has closed an $11 million seed financing to advance its pipeline of gene and cell therapies designed to preserve sight and extend ocular healthspan in patients with chronic blinding diseases.
Neuromyelitis optica spectrum disorder (NMOSD) is neuroinflammatory disease characterized by optic neuritis and myelitis with presence of anti-aquaporin-4 (AQP4) antibodies. Satralizumab is an anti-IL-6 receptor (IL-6R) humanized antibody approved for preventing relapses in NMOSD (AQP4 IgG positive) in adults and children in Japan.
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