Investigators from Proqr Therapeutics NV have tested their product QR-1011, an antisense oligonucleotide designed to correct splicing abnormalities within the ABCA4 gene that affect protein expression levels, in Stargardt disease.
Kriya Therapeutics Inc. has announced its gene therapy program for thyroid eye disease (TED), KRIYA-586. It is designed to be a one-time, adeno-associated virus (AAV) gene therapy that drives durable expression of a monoclonal antibody blocking the insulin-like growth factor 1 receptor (IGF-1R).
Diabetic retinopathy is the main diabetes complication affecting the eyes, and it leads to irreversible vision loss; oxidative stress and impairment of the retinal Muller cells are among the pathogenic mechanisms. Chinese researchers presented data on adiporon, an agonist of the adiponectin receptor protein 1 (ADPOR1) and ADIPOR2 that plays a crucial role in the metabolism of lipids and glucose, in preclinical mouse models of diabetes induced by streptozotocin.
Hangzhou Yirui Pharmaceutical Technology Co. Ltd. has described N-tetrazolyl aryl urea derivatives acting as bradykinin B1 receptor antagonists reported to be useful for the treatment of age-related macular degeneration, allergic conjunctivitis, pulmonary edema, neuralgia, infectious pneumonia, diabetic retinopathy, SARS-CoV-2 infection (COVID-19) and uveitis.
Putting into jeopardy what was on track to be the first approved therapy in Europe for geographic atrophy (GA), an advanced form of age-related macular degeneration that causes blindness, Apellis Pharmaceuticals Inc. learned of a negative trend vote by the EMA’s Committee for Medicinal Products for Human Use (CHMP) on its MAA for intravitreal pegcetacoplan.
Positive top-line data from Eyepoint Pharmaceuticals Inc.’s phase II study of EYP-1901 and the selective tyrosine kinase inhibitor vorolanib for treating wet age-related macular degeneration (AMD) is challenging Regeneron Pharmaceuticals Inc.’s recently approved Eylea (aflibercept).
A Character Biosciences Inc. patent describes polypeptides acting as low-density lipoprotein receptor (LDLR) ligands and reported to be useful for the treatment of age-related macular degeneration (AMD).
Inmed Pharmaceuticals Inc. has identified a lead cannabinol (CBN) analogue candidate, INM-089, to advance into additional in vivo studies for age-related macular degeneration (AMD).
As expected, Aldeyra Therapeutics Inc. received a complete response letter (CRL) from the U.S. FDA regarding its NDA for reproxalap in dry eye disease, with the agency requiring “at least one” additional study to prove efficacy of the reactive aldehyde species, or RASP, modulator.
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