Genetic mutations are the primary cause of most rare diseases. Although each condition affects a small fraction of the population, the global impact is significant, with an estimated 300 million individuals affected worldwide. A large proportion of pathogenic missense variants – estimated at 40%-60% – cause rare diseases by impairing protein stability. This underscores protein restoration as a promising therapeutic strategy. Read More

At the recent American Association for Cancer Research (AACR) Special Conference on Advances in Pancreatic Cancer Research, researchers from Cellectar Biosciences Inc. presented preclinical data on [225Ac]CLR-121225 (CLR-225), a novel actinium-based radio conjugate alpha-emitter for treatment in hypoxic pancreatic ductal adenocarcinoma. Read More

Cirrus Therapeutics Inc. has closed an $11 million seed financing to advance its pipeline of gene and cell therapies designed to preserve sight and extend ocular healthspan in patients with chronic blinding diseases. Read More

Cartography Biosciences Inc. has closed a $67 million series B financing to support its pipeline of T-cell engaging bispecific and multispecific antibody therapeutics that target novel and highly specific tumor antigens. Read More

OGG1 is a key enzyme in the base excision repair pathway, responsible for removing oxidized bases from DNA. In idiopathic pulmonary fibrosis (IPF), emerging evidence suggests that OGG1 also contributes to profibrotic gene expression, indicating a role beyond DNA repair. Read More

Mari Serebrov, BioWorld’s regulatory editor, shares how the publication’s 35-year commitment to fact-based, expert-driven journalism helps readers navigate complexity and make informed decisions. Read More

Cogent Biosciences Inc. has disclosed prodrugs and their active metabolites acting as GTPase KRAS and their mutant inhibitors reported to be useful for the treatment of cancer. Read More

Vitiligo is caused by autoreactive CD8+ T cells that react against skin melanocytes, where IL-15 has been shown to activate these T cells. Innovent Biologics (Suzhou) Co. Ltd. recently presented data on their monoclonal antibody (mAb) – IBI-3013 – that targets IL-15. IBI-3013 was evaluated in vitro in immune cell expansion and CD8 T-cell activation assays, as well as in vivo in a skin inflammation model and in a graft-vs.-host disease (GVHD) model. Read More

Vanqua Bio Inc. has synthesized C5a anaphylatoxin chemotactic receptor 1 (C5aR; CD88) antagonists reported to be useful for the treatment of autoimmune disease, cancer, infections, and cardiovascular, inflammatory and neurological disorders. Read More

Neuron23 Inc. has identified non-receptor tyrosine-protein kinase TYK2 inhibitors reported to be useful for the treatment of autoimmune disease, endocrine disorders, inflammatory disorders and multiple sclerosis. Read More

Idiopathic pulmonary fibrosis (IPF) is a rapidly progressing lung disease with high unmet needs and characterized by an excess in matrix deposition that leads to severe decline in lung functioning, where arginase expression and arginine metabolism seem to be involved and could be a promising target. Astrazeneca has presented data regarding their arginase inhibitor, AZD-8965, for the potential treatment of IPF. Read More

Dem Biopharma Inc. has divulged G-protein coupled receptor 84 (GPR84) agonists and phagocytosis inducers reported to be useful for the treatment of cancer, infections, neurological disorders and rheumatoid arthritis. Read More

Immuse Therapeutics Inc. has described E3 ubiquitin-protein ligase CBL-B (CBLB) inhibitors reported to be useful for the treatment of cancer. Read More

Bacterial resistance remains one of the biggest obstacles to antibiotic efficacy and spurs the constant search for next-generation antimicrobials. Oxacins can kill bacteria by inhibiting the activity of DNA gyrase. As potential next-generation oxacin-like drugs, researchers at Southwest University and collaborators have developed thiazolylcyanovinyl benzopyridone acids, among which the ethyl compound [I] turned out to be effective at killing several gram-negative and -positive bacterial strains in vitro as well as Klebsiella pneumonia in biofilms. It also eliminated wound infection in mice, without causing obvious off-target toxicity. Read More