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.
Recent findings discovered a mutation in the METTL23 gene, which encodes methyltransferase-like protein 23, in a pedigree of normal-tension glaucoma (NTG). The aim of researchers from the Institute for Vision Research, The University of Iowa Roy J and Lucille A Carver College of Medicine was to confirm an association of mutations in this gene with NTG.
Previous findings had shown that injecting pepatin-1 prevented the death of retinal ganglion cells (RGCs) in rats with ocular hypertension. Additionally, transcriptomic analysis in RGCs revealed cAMP response element-binding protein (CREB) signaling to be activated by peptain-1 conjugated with a cell-penetrating peptide, named P1-CPP.
Not long after Ocular Therapeutix Inc. unveiled positive phase I data in non-proliferative diabetic retinopathy with Axpaxli (axitinib intravitreal implant), Eyepoint Pharmaceuticals Inc. offered less-sunny phase II data from the Pavia trial testing Duravyu (vorolanib intravitreal insert), previously known as EYP-1901, in the same indication.
Schepens Eye Research Institute presented new preclinical data on its AAV2.sFasL gene therapy, an adeno-associated virus (AVV2) encoding soluble Fas ligand (sFasL) for the potential prevention of glaucoma.
Eluminex Biosciences Ltd. announced the FDA’s acceptance of their IND application for EB-105, a trispecific fusion antibody targeting vascular endothelial growth factor A (VEGF-A, and isomers), VEGF-B, placental growth factor (PlGF), angiopoietin-2 (Ang-2) and interleukin-6 receptor (IL-6R) for the treatment of diabetic macular edema (DME).
Revopsis Therapeutics Inc. has closed its first seed funding round, raising $16.5 million that will fund the completion of ongoing IND-enabling studies with its lead candidate, RO-104, for the treatment of neovascular age-related macular degeneration (nAMD). Developed using the company’s proprietary Rev-Mod platform, RO-104 is a potentially first-in-class fully human modular trispecific biologic.
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