Previous studies have linked variants of complement factor H (CFH) and its alternative splicing isoform, FHL-1, with increased complement activation and risk of age-related macular degeneration (AMD).
Ascidian Therapeutics Inc. recently provided preclinical data for ACDN-01, an AAV-encoded RNA exon editor targeting ABCA4, being developed for the treatment of ABCA4-related retinopathies, including Stargardt disease.
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
A new adeno-associated viral vector-driven CR2-CR1 fusion protein, named KRIYA-825, was tested in vivo in mice with sodium iodate-induced geographic atrophy.
Researchers from Tokyo Medical University presented data from a study that assessed microRNA (miRNA) profiles in extracellular vesicles (EVs) isolated from vitreous humor and serum of patients with ocular sarcoidosis and vitreoretinal lymphoma (VRL).
Eluminex Biosciences Ltd. has presented results from preclinical studies of EB-105, a humanized trispecific antibody targeting IL-6 receptor (IL-6R), vascular endothelial growth factors (VEGFs) and angiopoietin-2 (Ang-2) for the treatment of diabetic macular edema.
Raytone Biotechnology Co. Ltd. has described compounds based on oxidative stress and antiamyloid aggregations reported to be useful for the treatment of cataracts and presbyopia.
At the recently concluded ARVO meeting, Splicebio S.L. presented the first preclinical results on the company’s new candidate, SB-007, for the treatment of Stargardt disease, a rare genetic disease caused by mutations in the ABCA4 gene with no approved treatment on the market.
Vascular dysfunction that causes leakage of plasma proteins such as fibrinogen, fibrin deposits and innate immune cell activation is the cause of neurodegenerative ocular diseases, including diabetic macular edema, age-related macular degeneration or diabetic retinopathy.
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
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