There is still no effective vaccine or cure for HIV. Scientists are considering options ranging from longer-term antiretroviral therapy (ART) that space out injections by several years to long-lasting pre-exposure prophylaxis (PrEP) that acts as a vaccine while immunization is achieved. What else can be done? The “Innovations in HIV virology: Translating discoveries into novel therapies” symposium in basic science at the 13th IAS Conference on HIV Science (IAS 2025), which took place from July 13 to 17, 2025, in Kigali, Rwanda, showcased some of the new ideas that the scientific community are developing.
There is still no effective vaccine or cure for HIV. Scientists are considering options ranging from longer-term antiretroviral therapy (ART) that space out injections by several years to long-lasting pre-exposure prophylaxis (PrEP) that acts as a vaccine while immunization is achieved. What else can be done? The “Innovations in HIV virology: Translating discoveries into novel therapies” symposium in basic science at the 13th IAS Conference on HIV Science (IAS 2025), which took place from July 13 to 17, 2025, in Kigali, Rwanda, showcased some of the new ideas that the scientific community are developing.
Fragile X syndrome (FXS), the most common inherited cause of intellectual disability and autism, is caused by silencing of the Fmr1 gene, leading to a lack of the FMRP protein, which regulates protein synthesis in neurons. One key pathway affected by FMRP loss is the metabotropic glutamate receptor 5 (mGluR5) signaling pathway, where activation of mGluR5 leads to excessive translation of several proteins involved in synaptic plasticity.
Rhabdomyosarcoma is the most frequent cancer affecting soft tissues in children, and existing therapies often fail to stop relapse or prolong survival. Researchers in Germany and the U.K. have developed a potential new therapy based on natural killer cells expressing a chimeric antigen receptor against epidermal growth factor receptor (EGFR-CAR).
SK Bioscience Ltd. announced that the company has submitted an investigational new drug (IND) application to the South Korean Ministry of Food and Drug Safety.
In a recent study published in Nature Genetics, a team of scientists used CRISPR-Cas9 gene editing to systematically analyze genetic weaknesses in uveal melanoma cells and comprehensively map monogenic and digenic dependencies.
Gasherbrum Bio Inc. has patented new heterocyclic glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, among others.
Researchers have identified bi-allelic variants in the POPDC2 gene as the cause of a rare inherited cardiac syndrome characterized by sinus node dysfunction, atrioventricular (AV) conduction defects and hypertrophic cardiomyopathy.
Robert Williamson, CEO of Triumvira Immunologics, and Hernan Bazan, CEO of South Rampart Pharma, discuss bracing for tariffs, NIH grant cuts and gifting China a biopharma leadership position.
Incretin-based therapies have demonstrated substantial efficacy in the treatment of metabolic diseases such as obesity and type 2 diabetes but the need for frequent injections remains a major barrier to patient adherence and comfort.
While people living with HIV can lead virtually normal lives thanks to antiretroviral therapy (ART), HIV persists in a latent state within cellular reservoirs that scientists do not know how to eliminate. “Transcription is a critical step in the viral life cycle. … But there are currently no drugs...
Obesity and diabetes are known risk factors for sarcopenia. Insulin resistance that occurs during type 2 diabetes promotes impaired synthesis of muscle protein and muscle breakdown. Juvena Therapeutics Inc. has presented data at ENDO 2025 conference for JUV-161, a novel secreted protein that...
Acromegaly is a rare, chronic endocrine disorder characterized by excessive secretion of growth hormone (GH), most commonly resulting from a benign pituitary adenoma.
Discoidin domain receptor tyrosine kinase 1 (DDR1) contributes to tumor progression by promoting the alignment and densification of collagen fibers within the extracellular matrix (ECM), thereby facilitating the development of an immune-excluded tumor microenvironment (TME).
As of May 2023, the WHO recommends the use of a monovalent XBB.1 descendent lineage, such as omicron XBB.1.5, as the vaccine antigen for COVID-19 vaccines. However, BA.2.86 and its descendent lineages, such as JN.1, have emerged and rapidly spread worldwide.
Nipah (NiV) and Hendra (HeV) viruses, two closely related members of the Henipavirus genus, continue to raise global concern due to their high mortality rates and potential for zoonotic spillover. Despite repeated outbreaks, there are still no licensed antiviral treatments targeting these...
Amphix Bio LLC has been granted U.S. FDA orphan drug designation for its lead candidate AMFX-200 for the treatment of acute spinal cord injury (SCI). AMFX-200 is an FGFR (fibroblast growth factor receptor) and ITGB1 (integrin β1) agonist peptide amphiphile scaffold. In preclinical models of acute...
Voyager Therapeutics Inc. has expanded its Alzheimer’s disease (AD) pipeline with the addition of a wholly owned program that modulates the expression of apolipoprotein E (APOE). Using a proprietary intravenous-delivered, blood-brain barrier (BBB)-penetrant Tracer capsid, the product delivers a...
Illimis Therapeutics Inc. raised ₩58 billion (US$42 million) in a series B financing round. The funds will support development of ILM-01, its lead bispecific fusion protein candidate, into preclinical development for Alzheimer’s disease by the second half of 2025, along with the company’s...
Acumen Pharmaceuticals Inc. and JCR Pharmaceuticals Co. Ltd. have entered a collaboration, option and license agreement to develop a novel therapeutic candidate for the treatment of Alzheimer’s disease (AD).
Pfizer Inc. has identified signal transducer and activator of transcription 6 (STAT6) inhibitors reported to be useful for the treatment of Alzheimer’s disease, cancer, atherosclerosis, metabolic dysfunction-associated steatohepatitis, respiratory and dermatological disorders.
Stimulating the production of β cells in the pancreas of individuals with type 1 diabetes may be a way to renormalize their glucose metabolism. Researchers from Spain and the U.S. may have moved one step closer to this goal by stimulating formation of new β cells in the pancreas of a mouse model of...
Abdominal aortic aneurysm is a life-threatening cardiovascular disease characterized by chronic inflammation and irreversible dilatation of the abdominal aorta and is asymptomatic at the early stages of the disease, with diagnosis usually relying on imaging techniques and surgery being the main...
Hypertensive nephropathy is a major complication of hypertension characterized by inflammation and fibrosis in the kidneys. Recent findings have suggested that agrin may play a critical role in some tissue types such as in the heart or skeletal muscle.
The switch will be flicked today to make the world’s largest dementia-related proteomics dataset freely available to researchers, at the same time as members of the consortium which compiled it publish the proteomics signatures of major neurodegenerative diseases that they uncovered in a first...
A recent publication in Science Advances has uncovered NMNAT1 as a promising therapeutic target for alcohol-associated liver disease (ALD). Early ALD can be reversible, but prolonged alcohol abuse may lead to progressive steatohepatitis, fibrosis...
Toll-like receptor 3 (TLR3) has been noted to impact liver disease and promote liver inflammation. Researchers from Southern Medical University screened a natural product molecular library and found that ellipticine exerted moderate TLR3...
Researchers from Genentech Inc. have developed a novel single-domain antibody (VHH) targeting the IL-23 signaling pathway, which can be administered orally to effectively treat inflammatory bowel disease in animal models.
Is fecal microbiota transplant effective? Is it really safe? And is it really all the same? Scientists at the University of Chicago have investigated the regional differences in gut environments to question these interventions.
Researchers at Gachon University and Seoul National University Hospital have synthesized a disintegrin and metalloproteinase with thrombospondin motifs 4 (ADAMTS4; aggrecanase-1) inhibitors reported to be useful for the treatment of hepatic fibrosis.
Dice Molecules SV LLC has divulged integrin α4β7 (LPAM-1) antagonists reported to be useful for the treatment of graft-vs.-host disease, type 1 diabetes, inflammatory bowel disease, colitis and primary sclerosing cholangitis.
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.