Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular and Clinical Ophthalmology Basel and collaborators proposed a novel dual AAV vector system to deliver a split-intein adenine base editor for precise correction of the most common Stargardt mutation, c.5882G>A (p.Gly1961Glu).
Researchers from Columbia University Irving Medical Center published data from a study that assessed GiGA1, a selective G protein-gated inwardly rectifying potassium channel (GIRK) activator, in physiologically and clinically relevant ex vivo mouse models of tumor-associated and provoked seizures.
Dewpoint Therapeutics Inc. has nominated a second development candidate, DPTX-3496. The oral, small-molecule condensate modulator targets β-catenin to address Wnt-driven colorectal cancer, triple-negative breast cancer and non-small-cell lung cancer. IND-enabling studies have commenced, with an IND filing planned in the second half of this year.
Multiple organ dysfunction syndrome is a life-threatening condition derived from heat stress or sepsis, among other conditions, that can lead to irreversible organ damage and death if left untreated. Gasdermin (GSDMD) is an executor protein that causes cell lysis, facilitates the release of inflammatory mediators and is involved in pyroptosis, apoptosis and necroptosis processes leading to inflammatory cell death or PANoptosis.
Harness Therapeutics Ltd. has raised fresh financing to further develop its technology for upregulating the translation of mRNA into proteins, and in particular to take on a previously undruggable target in Huntington’s disease.
Omniamed Co. Ltd. and Pohang University of Science & Technology have patented compounds acting as nitric oxide (NO) scavengers and reported to be useful for the treatment of cancer, allergy, obesity, myelofibrosis, autoimmune diseases, inflammatory, cardiovascular and neurological disorders, among others.
Nanjing Synnocare Pharmaceutical Technology Co. Ltd. and Tianjin Synnocare Biomedical Technology Co. Ltd. have jointly developed new N-myristoyltransferase 1 (NMT1) inhibitors reported to be useful for the treatment of cancer, malaria, leishmaniasis, rhinovirus and HIV infections.
Work at Aston University has led to the identification of tissue transglutaminase (TGM2) inhibitors potentially useful for the treatment of fibrosis, thrombosis, cancer, AIDS, inflammation, transplant rejection, neurodegenerative and autoimmune diseases, among others.
Exelixis Inc. has synthesized fused pyrazole derivatives acting as ubiquitin carboxyl-terminal hydrolase 1 (USP1) inhibitors reported to be useful for the treatment of cancer.
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