A therapeutic strategy based on alternative splicing of the MECP2 gene could restore protein levels in Rett syndrome, a neurological disorder caused by mutations in that gene. Scientists at Baylor College of Medicine have successfully tested this approach both in vitro in neurons from Rett patients that produce some functional protein, correcting the altered gene expression and improving neuronal functions, and in vivo in mice. Read More
CD47, or integrin-associated protein, is a ubiquitously expressed cell surface protein that is overexpressed in many solid tumors and hematologic malignancies, including multiple myeloma, with higher levels associated with poor prognosis. In a recent study published in iScience, researchers from Westlake University (China) engineered an oncolytic vaccinia virus encoding an anti-mouse CD47 nanobody (OVV-aCD47nb) that combines direct oncolysis with localized CD47-SIRPα axis blockade. Read More
Antiverse Ltd. has announced the close of a $9.3 million series A financing. The funding will support expansion of the company’s proprietary AI antibody design platform, accelerate development of its internal therapeutic pipeline, and advance lead antibody programs toward in vivo efficacy studies. Read More
UCB SA and Antengene Corp. Ltd. have entered into a license agreement that grants UCB a worldwide exclusive license to further develop, manufacture and commercialize ATG-201. Read More
A new isoform of proliferating cell nuclear antigen (PCNA) – cancer-associated PCNA (caPCNA) – that is specifically expressed in cancer tissues has been reported. Because cancer cells and HIV-infected cells have similar features, researchers from City of Hope National Medical Center tested the anit-HIV effects of a small-molecule compound, AOH-1996, that targets caPCNA. Read More
The Korea Institute of Science and Technology (KIST) and Neocannbio Co. Ltd. have jointly identified compounds acting as nitric oxide (NO) production inhibitors with potential for the treatment of inflammatory bowel disease (IBD). Read More
Sinopia Biosciences Inc. has entered into a target discovery collaboration with Ono Pharmaceutical Co. Ltd. focused on a group of rare metabolic disorders with significant unmet medical need. Read More
Pfizer Inc. has discovered substituted pyridine compounds acting as sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors and thus described as potentially useful for the treatment of diabetes, chronic kidney disease, nonalcoholic fatty liver disease (NAFLD; MASLD), phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism-spectrum disorders, among others. Read More
Chengdu Cynogen Bio-Pharmaceutical Technology Co. Ltd. has synthesized CDK2/cyclin E1 inhibitors designed for use in the treatment of cancer. Read More
IRAK1, a key mediator of TLR/IL-1R signaling, drives tumor cell proliferation and a pro-inflammatory microenvironment when aberrantly activated. Its overexpression has been observed in multiple cancers, including hepatocellular carcinoma (HCC). Read More
Hangzhou Innogate Pharma Co. Ltd. and Innorace Biopharma Co. Ltd. have patented 20-hydroxyeicosatetraenoic acid (20-HETE) production inhibitors. They are reported to be useful for the treatment of cancer, obesity, diabetes, Alzheimer’s and Parkinson’s disease, osteoporosis, cardiovascular and cerebrovascular disorders, among others. Read More
Bromodomain-containing protein 4 (BRD4) is an epigenetic regulator that promotes transcription of oncogenic drivers such as MYC and contributes to tumor growth and therapy resistance in colorectal cancer, making it a promising therapeutic target. Researchers from Hangzhou Kexing Biochem Co. Ltd. described the discovery and preclinical characterization of compound [I], a potent BRD4 inhibitor. Read More
