Researchers from Broad Institute and Harvard University presented the discovery of all-in-one virus-like particles (VLPs) designed to deliver prime editor (PE) ribonucleoprotein (RNP) complexes into mammalian cells.
E-Therapeutics plc has offered a pipeline update, following the nomination of novel target genes, which have yielded promising results in preclinical studies.
N4 Pharma plc’s subsidiary Nanogenics Ltd. has signed a contract to start the formulation and sequence selection work to prepare its ECP-105 product for testing in preclinical studies.
Investigators from Proqr Therapeutics NV have tested their product QR-1011, an antisense oligonucleotide designed to correct splicing abnormalities within the ABCA4 gene that affect protein expression levels, in Stargardt disease.
Kriya Therapeutics Inc. has announced its gene therapy program for thyroid eye disease (TED), KRIYA-586. It is designed to be a one-time, adeno-associated virus (AAV) gene therapy that drives durable expression of a monoclonal antibody blocking the insulin-like growth factor 1 receptor (IGF-1R).
Diabetic retinopathy is the main diabetes complication affecting the eyes, and it leads to irreversible vision loss; oxidative stress and impairment of the retinal Muller cells are among the pathogenic mechanisms. Chinese researchers presented data on adiporon, an agonist of the adiponectin receptor protein 1 (ADPOR1) and ADIPOR2 that plays a crucial role in the metabolism of lipids and glucose, in preclinical mouse models of diabetes induced by streptozotocin.
Hangzhou Yirui Pharmaceutical Technology Co. Ltd. has described N-tetrazolyl aryl urea derivatives acting as bradykinin B1 receptor antagonists reported to be useful for the treatment of age-related macular degeneration, allergic conjunctivitis, pulmonary edema, neuralgia, infectious pneumonia, diabetic retinopathy, SARS-CoV-2 infection (COVID-19) and uveitis.
A Character Biosciences Inc. patent describes polypeptides acting as low-density lipoprotein receptor (LDLR) ligands and reported to be useful for the treatment of age-related macular degeneration (AMD).
Inmed Pharmaceuticals Inc. has identified a lead cannabinol (CBN) analogue candidate, INM-089, to advance into additional in vivo studies for age-related macular degeneration (AMD).
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