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BioWorld - Saturday, December 13, 2025
Home » Topics » Disease categories and therapies » Ocular

Ocular
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Diagram showing parts of the eye
Ocular

Sightstream’s MC1R agonist ameliorates corneal stromal edema in preclinical model

May 15, 2024
Annually in the U.S., about 40,000 people with corneal endothelial cell dystrophy receive a corneal transplant. The ligand of melanocortin MC1 receptor (MC1R) has been shown to protect the corneal endothelial cells from stress and injury in several models.
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Ocular

CTX-114, an engineered variant of FHL-1 with enhanced complement regulatory activity

May 15, 2024
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).
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Ocular

ACDN-01 results in robust, durable and well-tolerated ABCA4 exon editing in preclinical models

May 15, 2024
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.
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Vial and syringe with DNA
Ocular

ASGCT: ‘From darkness to light’ in ocular gene therapy

May 14, 2024
By Mar de Miguel
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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Mouse retina.
Ocular

KRIYA-825 ameliorates retinal degeneration in mice, study shows

May 14, 2024
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.
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3D rendering of exosomes and vesicles
Biomarkers

miRNA profiles in extracellular vesicles differentiate between ocular sarcoidosis and vitreoretinal lymphoma

May 13, 2024
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).
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Close up of man's eye
Ocular

Preclinical data on trispecific fusion antibody EB-105 presented

May 13, 2024
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.
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Ocular

Raytone Biotechnology discovers new compounds to treat cataracts and presbyopia

May 10, 2024
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.
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Eye and DNA
Ocular

Preclinical safety and efficacy of intein-based dual-AAV gene therapy for Stargardt disease

May 10, 2024
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.
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Eye wireframe illustration
Ocular

Therini Bio’s THN-391 shows efficacy in rodent models of ocular diseases

May 10, 2024
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.
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