Researchers at the University of Sydney have uncovered a mechanism that may explain why glioblastoma returns after treatment, and the world-first discovery offers new clues for future therapies. Glioblastoma is one of the deadliest brain cancers, accounting for about half of all brain tumors, with a median survival rate of just 15 months. Despite surgery and chemotherapy, more than 1,250 clinical trials over the past 20 years have struggled to improve survival rates.
Researchers at the University of Sydney have uncovered a mechanism that may explain why glioblastoma returns after treatment, and the world-first discovery offers new clues for future therapies. Glioblastoma is one of the deadliest brain cancers, accounting for about half of all brain tumors, with a median survival rate of just 15 months. Despite surgery and chemotherapy, more than 1,250 clinical trials over the past 20 years have struggled to improve survival rates.
During aging, hematopoietic stem cells (HSCs) undergo functional decline affecting their ability to regenerate the hematopoietic system and support lymphoid cell production. This functional decline contributes to some aging-related diseases. Moreover, aging is associated with biomechanical changes in HSCs, including alterations in nuclear envelope tension and nuclear mechanical integrity and mechanotransduction. However, it remains unclear whether aging of somatic stem cells can be prevented by targeting changes in nuclear mechanosignaling.
Viatris Inc. has announced FDA clearance of its IND application for MR-146, an Enriched Tear Film (ETF) AAV gene therapy candidate for the treatment of neurotrophic keratopathy. The company plans to initiate a phase I/II trial in patients with neurotrophic keratopathy in the first half of next year.
Werewolf Therapeutics Inc. has announced that its priorities for the coming year include a focus on the company’s Inducer T-cell engager platform, where preclinical studies have demonstrated robust silencing and reduction of off-tumor toxicity.
Researchers from Chimerix, now part of Jazz Pharmaceuticals, presented preclinical data on ONC-206, a compound that functions as both an agonist of the mitochondrial protease Caseinolytic peptidase P (CLPP) and an antagonist of the G protein-coupled receptor DRD2, in models of triple-negative breast cancer (TNBC).
Cartography Biosciences Inc. has received IND approval from the FDA for CBI-1214, a T-cell engager being developed for the treatment of colorectal cancer.
Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has disclosed RAC-α serine/threonine-protein kinase (AKT1; PKB α) (Glu17Lys mutant) inhibitors reported to be useful for the treatment of leukemia.
The opioid crisis may not be front and center anymore, but it’s raging still. Elysium Therapeutics Inc. CEO Greg Sturmer talks about his firm’s candidate for a solution to the medical and societal problem.
Most patients with myelodysplastic syndrome (MDS) exhibit variable degrees of anemia due to impaired erythropoiesis. Ameliorating anemia and reducing the dependence on transfusion may enhance the quality of life of these patients and improve their survival rates.
Although hormone receptor-positive (HR+) breast cancer accounts for over 70% of cases, 20%-30% of patients still experience relapse despite endocrine therapies such as tamoxifen. Recurrence is also observed in HER2+ breast cancer, even with HER2-targeted antibodies and antibody-drug conjugates...
Modex Therapeutics Inc. has developed MDX-2003, a first-in-class tetraspecific T-cell engager targeting CD19 and CD20 on B cells, while co-engaging CD3 and CD28 on T cells.
Researchers from Aera Therapeutics Inc. reported the preclinical profile of AERA-109, a novel, targeted in vivo CAR T therapy designed to treat B-cell-mediated autoimmune diseases.
Eilean Therapeutics LLC has announced it is advancing ZE74-0282 into first-in-human development, with the submission of an IND application and enrollment planned to begin in the first quarter of next year.
Advances in antiretroviral therapy (ART) now allow people living with HIV to lead normal lives with undetectable and nontransmissible levels of the virus in their blood. Yet that reality is limited to those with access to treatment. More than 40 million people worldwide live with HIV, with over a...
Nchroma Bio Inc. has received a certificate for clinical trial in Hong Kong to initiate a first-in-human phase I/II trial of CRMA-1001 for the treatment of chronic hepatitis B virus (HBV) infection. Dosing is expected to begin early next year.
Orthoflaviruses such as dengue, West Nile and Zika viruses are a threat to public health for which no specific treatments exist. Their protease NS2B-NS3, also called orthoflavivirin, is an attractive drug target because it is essential for virus maturation. Targeting viral proteases has already...
Tonix Pharmaceuticals Holding Corp. has licensed exclusive worldwide rights to TNX-4900 (formerly PW-507) from Rutgers University. TNX-4900 is a highly selective, small-molecule sigma-1 receptor (S1R) antagonist, which has demonstrated analgesic activity in multiple models of neuropathic pain.
Superoxide dismutase 1 (SOD1) mutations were among the first genetic causes identified in familial amyotrophic lateral sclerosis (ALS) and confer a toxic gain-of-function that drives motor neuron degeneration via protein misfolding, oxidative stress, mitochondrial dysfunction and neuroinflammation.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder marked by progressive degeneration of upper and lower motor neurons, resulting in paralysis and death typically within 3-5 years of symptom onset. Historically, treatment options have been extremely limited. However, the...
Researchers from Wuhan Institute of Biomedical Sciences, Jianghan University and collaborators recently showed that activating the endogenous transcription factors Ngn2 and Isl1 via CRISPRa can directly reprogram astrocytes into functional motor neurons in the mouse spinal cord.
Epilepsygtx Ltd. has raised a $33 million series A to fund a phase I/IIa trial of EPY-201, a gene therapy for treating drug-resistant focal epilepsy. EPY-201 uses an adeno-associated viral vector to deliver KCNA1, the gene encoding Kv1.1, a potassium ion channel that modulates neuronal excitability.
Rona Therapeutics Co. Ltd. has announced the submission of RN-5681 to the Australian Human Research Ethics Committee (HREC), and anticipates dosing to begin in a phase I trial in the first quarter of next year.
In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences and Xi’an Jiao Tong University Health Science Center found that Mesothelin (MSLN) was significantly elevated in rheumatoid arthritis (RA)...
It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its half a million participants.
South Korean researchers led by Lee In-suk of Yonsei University have reported the most complete oral microbiome catalog to date, with more than 72,000 genomes. Detailed in Cell Host & Microbe on Nov. 12, 2025, the database is expected to serve as a universal platform for academia and enable...
Yes-associated protein (YAP), encoded by Yes1-associated transcriptional regulator (YAP1) and transcriptional coactivator with PDZ-binding motif (TAZ), encoded by WW domain containing transcription regulator 1 (WWTR1), are two crucial paralog...
Researchers from the Medical School of Nanjing University hypothesized that in ulcerative colitis, the gut-resident macrophages may be compromised, leading to impaired integrity of the epithelial barrier. “From a basic science standpoint, our work...
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH)...
The U.K. Medicines and Healthcare products Regulatory Agency is calling for unified standards to harmonize microbiome research, after revealing major inconsistencies in the results when labs around the world analyzed identical reference samples of...
Samjin Pharmaceutical Co. Ltd. has disclosed inhibitors of 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) and/or estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) and/or HSD17B2 (17β-HSD2). As such, they are believed to be...
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.
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