A new multi-omics approach to unpicking how noncoding gene variants influence the development of common chronic diseases has identified tens of thousands of instances where variants have an impact on gene expression levels and gene splicing, the post-transcriptional modification that allows one gene to code for multiple proteins. Read More

Ischemic stroke is a fatal condition caused by an arterial embolism that blocks the blood flow through the cerebral artery, frequently being a cause of mortality and disability. Fibroblast growth factor 21 (FGF21) is likely the only member of the FGF family that may cross the blood-brain barrier. Among its functions, inflammatory regulation, energy metabolism, vascular homeostasis, oxidative stress and tissue repair can be highlighted. Read More

Aprea Therapeutics Inc. has entered into a material transfer agreement with MD Anderson Cancer Center that will support investigation of APR-1051 as a potential treatment for head and neck squamous cell carcinoma (HNSCC). Read More

Harbour Biomed Ltd. has announced the launch of Élancé Therapeutics with an aim to improve the treatment of obesity. Élancé seeks to address challenges in obesity treatment, by increasing overall body weight loss and fat mass loss, while preserving and even increasing muscle and lean mass. Read More

Inatherys SAS recently presented preclinical data for INA-03, an anti-transferrin receptor (TfR1/CD71) antibody-drug conjugate (ADC), being developed for the treatment of triple-negative breast cancer (TNBC). INA-03 was constructed by conjugating an antimitotic payload (MMAE) to a humanized IgG4 anti-human CD71 antibody using a novel valine-citrulline linker. Read More

Pierre Fabre SA and Redridge Bio AG have signed an exclusive R&D collaboration and license agreement to identify and develop biparatopic antibody drug candidates against multiple targets, with a focus on precision oncology, dermatology and rare diseases. The agreement provides for participation by Pierre Fabre in Redridge’s series A financing, as well as up-front, milestone and future sales royalty payments. Read More

Scientists at Dongguk University and Korean Research Institute of Bioscience and Biotechnology have disclosed benzofuran compounds targeting heterogeneous nuclear ribonucleoprotein A2/B1 (hnRNPA2B1) acting as RAF proto-oncogene serine/threonine-protein kinase (RAF-1; c-Raf) inhibitors reported to be useful for the treatment of cancer. Read More

Prostate cancer (PC), often referred to as a cold tumor, typically presents a poor immune response, making it difficult for immune-based therapies to be effective. Metastatic castration-resistant PC (mCRPC) presents a highly aggressive phenotype often refractory to systemic treatment. Read More

Laekna Pharmaceutical Ningbo Co. Ltd. has synthesized phosphatidylinositol 3-kinase α (PI3Kα) and its mutant (H1047R) inhibitors reported to be useful for the treatment of breast cancer. Read More

Researchers from the University of Barcelona and collaborators reported the discovery and preclinical characterization of novel imidazoline I2 receptor ligands (I2-IRs). Among the series, the most potent compound, [I], had a Ki value of 1.05 nM for the I2-IR and no I2/α2 selectivity. Read More

Sagimet Biosciences Inc. has obtained IND clearance from the FDA for TVB-3567 (ASC-60), a selective small-molecule fatty acid synthase (FASN) inhibitor set to enter clinical development for the treatment of acne. A first-in-human phase I trial of TVB-3567 in individuals with or without acne is expected to begin this year. Read More

Université Laval has identified PBRM derivatives acting as estradiol 17β-dehydrogenase 1 (HSD17B1; 17β-HSD1) inhibitors reported to be useful for the treatment of cancer. Read More

Katalytic Therapeutics Inc. has divulged proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to a phosphodiesterase PDE4-targeting moiety reported to be useful for the treatment of arthritis, Behçet’s disease, chronic obstructive pulmonary disease, non-small-cell lung cancer, inflammatory disorders, inflammatory bowel disease, psoriasis and rheumatoid arthritis. Read More

Pathogenic variants in the GJB2 gene are the most common genetic cause of congenital sensorineural hearing loss and are mostly associated with an autosomal recessive non-syndromic deafness 1A (DFNB1A). Read More

Lucy Therapeutics Inc. has described adenosine receptor agonists reported to be useful for the treatment of Rett syndrome and chronic heart failure. Read More