Alzheimer’s disease (AD) is a neurodegenerative condition in which amyloid plaques and neurofibrillary tangles accumulate in the brain. In addition to genetic factors, DNA damage and epigenetic alterations also play a key role in the pathogenesis and progression of this disease, altering gene expression, the functioning and maintenance of brain cells. DNA double-strand breaks (DSBs) and chromatin accessibility are two hallmarks of AD whose study could reveal new ways of approaching this disease.
At the Congress of the European Academy of Neurology, researchers from Dianthus Therapeutics Inc. presented preclinical data on DNTH-103, a monoclonal antibody engineered to selectively target the active form of complement C1s.
Alzheimer’s disease (AD) is a neurodegenerative condition in which amyloid plaques and neurofibrillary tangles accumulate in the brain. In addition to genetic factors, DNA damage and epigenetic alterations also play a key role in the pathogenesis and progression of this disease, altering gene expression, the functioning and maintenance of brain cells. DNA double-strand breaks (DSBs) and chromatin accessibility are two hallmarks of AD whose study could reveal new ways of approaching this disease.
At the Congress of the European Academy of Neurology, researchers from Dianthus Therapeutics Inc. presented preclinical data on DNTH-103, a monoclonal antibody engineered to selectively target the active form of complement C1s.
Hypochondroplasia (HCH) is a skeletal dysplasia similar to achondroplasia (ACH) but with milder features, that affects particularly the ossification of proximal long bones of arms and legs. Around 70% to 80% of cases of HCH are caused by N540K alterations in the FGFR3 gene. At the recent 6th Annual Achondroplasia & Skeletal Dysplasia Research Conference, researchers from Tyra Biosciences Inc. presented preclinical proof-of-concept data of TYRA-300, an oral FGFR3-selective inhibitor in a model of HCH.
Thirtyfivebio Ltd. has announced the award of a £643,371 (US$817,000) grant by Innovate UK to support work on first-in-class small-molecule inhibitors of G protein-coupled receptor 35 (GPR35).
Captor Therapeutics Inc. has disclosed proteolysis targeting chimeras (PROTACs) comprising an E3 ubiquitin ligase-binding moiety covalently linked to an induced myeloid leukemia cell differentiation protein MCL-1-targeting moiety through a linker.
Metabolic dysfunction-associated steatotic liver disease (MASLD) has a prevalence of about 25% in the adult population, with steatosis present in >5% of hepatocytes, hepatocyte ballooning and fibrosis as the main hallmarks. Scientists have tested effects of the galectin-3 inhibitor modified citrus...
A group of researchers in India have looked into the role of estrogen-related receptor γ (ERRγ) in the progression of colorectal cancer (CRC), and studied the effect of combination therapy with an ERRγ inverse agonist, DN-200434, and the AKT inhibitor MK-2206 in in vitro and in vivo assays.
Targeting monoacylglycerol O-acyltransferase 2 (MGAT2), an enzyme highly expressed in the small intestine, has been validated as an antiobesity strategy in the preclinical setting.
Biolexis Therapeutics Inc. is working on developing orally available, small-molecule AMPK activators that have been found to stimulate AMPK activity as well as dose-dependently enhance glucose uptake in human tissue.
Exsilio Therapeutics emerged from stealth mode on June 25, 2024, with $82 million from a series A financing that was co-led by Novartis Venture Fund and Delos Capital. The company plans to use naturally occurring, mobile genetic elements to integrate therapeutic genes at a defined location in the...
Tonix Pharmaceuticals Holding Corp. has been awarded up to $34 million over 5 years under a contract with the U.S. Department of Defense (DoD)’s Defense Threat Reduction Agency (DTRA).
Shigella flexneri is the leading cause of endemic diarrhea and dysentery in some countries with low-to-middle incomes. There is still a lack of a licensed vaccine against this pathogen.
Researchers at The Scripps Research Institute and the IAVI Neutralizing Antibody Center are developing a novel experimental vaccine targeting the germline to stimulate precursor B cells and produce broadly neutralizing antibodies (bnAbs) against the membrane-proximal external region (MPER) of the...
Spur Therapeutics Ltd., formerly Freeline Therapeutics, has announced new data from its GBA1 Parkinson’s disease research program. In a subset of Parkinson’s disease patients with mutations in the GBA1 gene, such mutations lead to a deficiency of the glucocerebrosidase (GCase) enzyme and the...
Nurexone Biologic Inc. has announced a preclinical study to explore the potential of the company’s exosome-based therapies for regenerating damaged optic nerves. The study is led by principal investigators from the Sheba Medical Center Eye Institute.
Kbio Inc. has obtained IND clearance from the FDA for EV68-228-N, a human monoclonal IgG1 against the capsid of enterovirus D68 (EV-D68) designed as an intravenous therapeutic for the treatment of acute flaccid myelitis.
“Do dreams predict the future?” Abidemi Otaiku asked his audience at the 10th Congress of the European Academy of Neurology, currently being held in Helsinki. Most audience members, being hard-boiled scientists, did not believe they did. But Otaiku, whose work won the award for best clinical...
Medipal Holdings Corp. and JCR Pharmaceuticals Co. Ltd. have announced the completion of the clinical trial notification review by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for a phase I/II study of JR-446 for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo...
There is growing evidence of the role of soluble endoglin in the biology of platelets, including thrombosis. French researchers have investigated the role of genetic variants in the gene encoding endoglin, ENG, and the risk of venous thrombosis development.
Angiogenesis plays a crucial role in multiple myeloma and its prognosis, with the success of antiangiogenic therapies being limited. Exosomes mediate cell-to-cell cross talk during the progression of cancer by transporting their molecular cargo, which include long non-coding RNAs (lncRNAs); their...
Inflammatory bowel disease (IBD) is sometimes associated with spondyloarthritis (SpA) and it highly impacts patients’ quality of life. It is crucial to understand the pro-inflammatory processes that take place during the pathogenesis of IBD-associated SpA.
Snipr Biome ApS has received funding from the Bill & Melinda Gates Foundation to develop a microbiome-directed intervention designed to improve environmental enteric dysfunction by reducing gut entero-pathogen burden in pregnant women from low-...
Monte Rosa Therapeutics Inc. has submitted an IND application to the FDA for MRT-6160, a highly selective and orally bioavailable molecular glue degrader directed against VAV1 in development for systemic and neurological autoimmune diseases.
Resolution Therapeutics Ltd. has received approval from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study of its lead candidate RTX-001 in patients with decompensated liver cirrhosis.
The search for an effective therapy for liver fibrosis continues. A recent study by researchers at Medical University of South Carolina and collaborators explored the potential of a new compound, LP-340, as a treatment for liver fibrosis.
Enveda Biosciences (Enveda Therapeutics Inc.) has announced a new series B2 financing round of $55 million. The drug discovery and development company uses artificial intelligence (AI)-powered technologies to translate nature into new medicines.
Abbvie Inc. and Futuregen Biopharmaceutical (Beijing) Co. Ltd. have signed a license agreement to develop FG-M701, a next-generation TL1A antibody for the treatment of 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.