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.
Crispr Therapeutics AG has expanded its in vivo pipeline with two new programs, which utilize lipid nanoparticle (LNP)-based delivery of CRISPR/Cas9 gene-editing cargo to the liver.
Chronic granulomatous disease (CGD) is an immunodeficient disorder that is caused by mutations in genes that encode proteins of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase enzyme complex.
Researchers from Xiangya Hospital of Central South University published data from a study that explored potential treatment targets for cutaneous squamous cell carcinoma (cSCC) by investigating the regulatory mechanisms of follistatin-like 1 (FSTL1), which has been shown to be implicated in a variety of cancers.
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.
Maze Therapeutics Inc. has described sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of phenylketonuria, metabolic syndrome, chronic kidney disease and metabolic diseases.
Researchers at Biogen Inc. and Broad Institute Inc. have divulged glycogen synthase kinase-3α (GSK3A; GSK-3α) inhibitors reported to be useful for the treatment of cancer, neurological and metabolic diseases.
Norroy Bioscience Co. Ltd. has identified conjugates comprising fibroblast activation protein α (FAP)-targeting agents covalently linked to cytotoxic drugs or unlabeled or radiolabeled imaging agents through a linker acting as FAP ligands reported to be useful for diagnosis and treatment of cancer expressing FAP.
RSS
