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.
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).
By isolating a single clone from human isolates, researchers from the National Taiwan University College of Medicine aimed to enhance the immunogenicity of the Zika virus (ZIKV).
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.
Killer immunoglobulin-like receptor (KIR)-chimeric antigen receptor (CAR) T-cell therapies have previously demonstrated superior performance and functional persistence in solid tumor models, and the mesothelin-specific KIR-CAR T cells, Synkir-110, are now being evaluated in phase I trials by Verismo Therapeutics Inc.
Researchers from Indiana University presented data from a study that aimed to assess the therapeutic potential of interleukin-9 (IL-9) blockade in the context of lung cancer.
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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