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.
Homerun success of Novo Nordisk A/S’ semaglutide, which recently became the U.S.’s biggest blockbuster drug, is serving as an “inflection point” for obesity therapeutics and fueling the drive for new and improved therapies, speakers said at Bio Korea 2024 on May 8.
As the average cost of new drug R&D continues to skyrocket, the perception around using artificial intelligence (AI) as a tool to boost drug discovery is changing. “Developing new AI-based drugs is a difficult task, not only for Korea but also for countries with leading AI technology,” Hyeyun Jung, principal researcher of Korea Health Industry Development Institute’s Center for Health Industry Policy, told the audience at the Bio Korea meeting on May 9. “But there is a change in perception; [namely that] applying AI to new drug development is not an option but a necessity.”
Data from the transcatheter valve and vessel trial showed that percutaneous interventions in patients with aortic stenosis and coronary disease resulted in significantly lower mortality rates than those receiving surgical treatment.
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.
Macrophage colony-stimulating factor 1 receptor (CSF-1R) is a transmembrane tyrosine kinase receptor expressed in brain microglia, and mutations in the CSF1R gene have been linked to adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).
At last week’s ASGCT meeting, Adicet Bio Inc. presented a new CAR T-cell therapy, ADI-270, for the treatment of CD70-expressing tumors. ADI-270 uses CD27 as the binding domain and 4-1BB co-stimulatory domains plus CD3.
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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