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.
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.
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.
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).
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.
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.
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.
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.
Billion-dollar biopharma mergers fall short of the dream: Karen Carey, BioWorld’s senior managing editor and chief analyst, crunched the numbers on 21 of the biggest M&As and found very few have been, so far, good deals for the buyer.
At the 2025 meeting of the Conference on Retroviruses and Opportunistic Infections (CROI) it was the best of times, it was the worst of times. On the first full day of the conference, reports from the first HIV cure trial conducted in Africa, the RIO trial and others showed that perhaps, a broadly...
Researchers from Diosynvax Ltd. presented data from a study that aimed to evaluate the efficacy of a pan-H5 vaccine designed using computational biology and being developed for protection against avian influenza.
Atopic dermatitis (AD) is a chronic, inflammatory skin condition driven by complex immune mechanisms involving T cells. In the context of AD, OX40, a costimulatory receptor present on activated T cells, supports the function of inflammatory T cells, exacerbating skin dysfunction. Researchers from...
Antibody-drug conjugates, a well-established antitumor strategy, are used for targeted delivery of cytotoxic drugs through antibodies. As cancer research progresses, other technologies with different mechanisms are advancing in the oncologic pipeline. Researchers from Cybrexa Therapeutics Inc....
Scientists at Southern University of Science and Technology in Shenzhen, China, have identified a new regulator of the mammalian target of rapamycin (mTOR) pathway associated with the growth of colorectal cancer.
At the 2025 meeting of the Conference on Retroviruses and Opportunistic Infections (CROI) it was the best of times, it was the worst of times. On the first full day of the conference, reports from the first HIV cure trial conducted in Africa, the RIO trial and others showed that perhaps, a broadly...
Researchers from Shaanxi Panlong Pharmaceutical Group Co. Ltd., Shaanxi Pioneer Biotech Co. Ltd. and affiliated organizations presented the discovery and preclinical characterization of novel pleuromutilin antibiotics, designed as organ-targeted, multimechanism antimicrobial agents using organic...
Merck Sharp & Dohme LLC has synthesized viral replication inhibitors reported to be useful for the treatment of respiratory syncytial virus and metapneumovirus infections.
Neurodevelopmental disorders related to protein phosphatase 2A (PP2A) have been recently renamed as Houge-Janssens syndrome and they are caused by heterozygous, de novo pathogenic genetic variants in the PPP2R5D, PPP2R1A or PPP2CA genes. The syndrome is characterized by features such as...
For the first time, researchers have identified that inflammation – long associated with multiple sclerosis (MS) – appears to cause increased mutations that damage neurons linked to MS progression. Researchers at the Florey Institute and the University of Melbourne studied MS brain lesions, which...
Trimtech Therapeutics closed a £25 million (US$31 million) oversubscribed seed funding round to advance its targeted protein degradation treatments for neurodegenerative and inflammatory diseases.
Ribopeutic Inc. has divulged nucleic acid splicing modulators (particularly, Huntingtin [HTT; HD] [mutant] and/or transcriptional activator Myb [c-Myb]) reported to be useful for the treatment of cancer and Huntington’s disease.
Fragile X syndrome (FXS) is the most common inherited form of intellectual disability and a leading monogenic cause of autism, yet effective treatments remain elusive. Previous work showed that N-methyl-D-aspartate receptors (NMDARs) play a prominent pathophysiological role in FXS and other...
Endocannabinoids are lipid mediators that interact with G protein-coupled receptors, including cannabinoid CB2 receptor (CB2R), which is mainly expressed in peripheral tissues with immune functions.
Esophageal cancer, particularly esophageal squamous cell carcinoma (ESCC), is one of the most prevalent digestive malignancies, with a 5-year survival rate of only 20%. Although some cancer immunotherapies based on immune checkpoint inhibitors (ICIs) have shown promise for ESCC, only a minority of...
Urachal carcinoma is a rare cancer which lacks a standard drug therapy, and for which knowledge regarding its immunohistochemical features remains unclear. The aim of a recently reported investigation was finding potential markers and targets for urachal carcinoma based on antibody-drug conjugate...
Prostate cancer is still one of the main causes of cancer death among men; while prostate-specific antigen (PSA) testing is done for screening, there are recommendations against its use due to its nonspecific and suboptimal results. There is thus an urgent need for new biomarkers that are more...
Exposure to high-dose radiation can result in gastrointestinal (GI) acute radiation syndrome, which is characterized by disruption of the mucosal layer, intestinal epithelial barrier dysfunction, and abnormal inflammatory responses. Currently,...
Researchers from Ileadbms Co. Ltd. presented the discovery and preclinical characterization of IL-21120033, a new CXCR7 agonist being developed for the treatment of inflammatory bowel disease.
Neurenati Therapeutics Inc.’s NEU-001 has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA for the treatment of Hirschsprung disease.
Researchers from CJ Bioscience Inc. presented the discovery and preclinical characterization of CJRB-201, a novel microbiome-based therapy for the treatment of inflammatory bowel disease (IBD).
Scientists from the Lebanese American University investigated the role of acetyl-CoA synthetase short chain family member 2 (ACSS2) in inflammatory bowel disease, including ulcerative colitis and Crohn’s disease.
Researchers from Sunrock Biopharma SL presented preclinical data on SRB-1, a CCR9-depleting antibody aimed to restore immune homeostasis in patients with inflammatory bowel disease.
Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.