https://www.bioworld.com/rss en-us Whether by fine-tuning neurotransmitter signaling or silencing disease-associated genes, emerging biologic therapies are reshaping neuroscience drug development, according to presentations at the FENS Forum 2026. http://www.bioworld.com/articles/732497 Thu, 09 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732497-fens-2026-next-gen-biologics-take-on-brain-disease Silhouette with keyhole Chronic liver disease and hepatocellular carcinoma (HCC) associated with chronic hepatitis B virus (HBV) are serious health problems in highly endemic areas. Investigators from the National Health Research Institutes have developed a protein-based recombinant vaccine, rHBx-rHBc149, for this unmet medical need. http://www.bioworld.com/articles/732485 Wed, 08 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732485-recombinant-vaccine-inhibits-hbv-associated-hcc-growth Liver with hepatitis viruses, blood cells At the recently opened FENS Forum 2026 in Barcelona – the Federation of European Neuroscience Societies’ flagship congress and Europe’s largest neuroscience meeting – a symposium on ectodomain shedding showcased how soluble synaptic proteins are emerging as both biomarkers and therapeutic candidates for disorders ranging from autism to schizophrenia. http://www.bioworld.com/articles/732479 Wed, 08 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732479-fens-2026-shedding-light-on-the-sheddome Art concept for medical research Myelin is crucial for the correct functioning of nerve signaling and its loss is a feature of neurological disorders. The cells responsible for myelination in the CNS are oligodendrocytes (OLs), which derive from oligodendrocyte precursor cells (OPCs). http://www.bioworld.com/articles/732457 Tue, 07 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732457-gde4-blockade-promotes-remyelination-study-finds Myelin sheath and neurons in the brain Myelin sheath and neurons in the brain Metachromatic leukodystrophy (MLD) is a rare inherited lysosomal storage disorder characterized by progressive neurodegeneration resulting from loss of arylsulfatase A (ARSA) activity. Researchers at Kazan Federal University reported preclinical efficacy data for a gene therapy candidate in a porcine model of MLD. http://www.bioworld.com/articles/732453 Tue, 07 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732453-aavolig001-arsa-shows-promise-in-preclinical-mld-study Art concept for gene therapy research Separate research teams have reported new insights into resistance mechanisms to the antibody-drug conjugate (ADC) Padcev (enfortumab vedotin, Astellas Pharma Inc./Pfizer Inc.), and possibly to ADCs more broadly. Urothelial cancer drug Padcev, which targets the cell adhesion molecule Nectin-4, was approved in 2019 and is currently one of Pfizer’s top 10 medicines and vaccines, generating $1.94 billion in 2025. http://www.bioworld.com/articles/732450 Tue, 07 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732450-target-high-but-uptake-defective-state-identified-in-adc-resistance 3D rendering of an antibody-drug conjugate Ovarian clear-cell carcinoma is a cancer characterized by inherent resistance to platinum-based therapies and lacks an effective targeted therapeutic approach. On the other hand, chaperone-mediated autophagy is a lysosomal degradation mechanism to stabilize cellular homeostasis during stressful scenarios, but its role in modulating platinum response in ovarian cancer is not well understood. http://www.bioworld.com/articles/732400 Mon, 06 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732400-lamp2a-modulation-regulates-ovarian-cancer-response-to-platinum Cancer cell destruction by nanoparticles Researchers in Korea have investigated the interaction of SET-domain containing 1B (SET1B) with other proteins in vitro, as well as the impact of SET1B depletion in hepatocellular carcinoma cell lines. http://www.bioworld.com/articles/732398 Mon, 06 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732398-set1b-blockade-as-an-approach-in-hepatocellular-carcinoma Illustration of tumor in the liver The majority of epilepsies are developmental disorders that start in childhood. But there is a large minority that starts in late adulthood. And increasingly, researchers are suspecting that such epilepsies share mechanisms with dementia. Summarizing the highlights of epilepsy research presented at the recent Annual Congress of the European Academy of Neurology (EAN), Aleksandar Ristic told his audience that the biggest epilepsy story out of the Congress was “not a drug, but it was a reframing.” http://www.bioworld.com/articles/732395 Mon, 06 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732395-hyperexcitability-may-be-joint-problem-in-epilepsy-dementia Illustration of brain and brain waves, concept of focal seizure Human biology is extraordinarily complex, and that sophistication emerges from the very beginning. During embryonic and fetal development, the organism’s architecture is shaped through the organization of tissues, the establishment of molecular pathways, and the coordination of signals that will later sustain the body as an integrated system. It is likely the most delicate stage of life, where any disturbance in that foundational process can have lasting consequences on health. http://www.bioworld.com/articles/732374 Fri, 03 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732374-cinp-2026-prenatal-and-adolescent-stress-shape-brain-vulnerability At the 2026 World Congress of Neuropsychopharmacology (CINP), held in Glasgow June 26-29, 2026, researchers from Japan’s National Center of Neurology and Psychiatry (NCNP) showcased how human organoid technologies are reshaping the study of neurodevelopmental vulnerability, addiction and psychiatric disorders. http://www.bioworld.com/articles/732329 Wed, 01 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732329-cinp-2026-organoids-reveal-autism-and-addiction-mechanisms AI-generated image for brain organoid neural research Deep brain stimulation (DBS) through implanted electrodes has enabled fundamentally new ways of treating certain disorders. More than 100,000 severely ill patients have received an implant to treat Parkinson’s disease, which is DBS’ greatest success story. http://www.bioworld.com/articles/732241 Tue, 30 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732241-deep-brain-stimulation-from-the-shallows-tomorrows-bci-technology Black wavy lines forming an abstract sound wave.png The gut microbiota may be altered in people with depression as a result of treatment. These microorganisms reorganize differently in individuals who respond to therapy. In a multiomics study of antidepressant-naive patients presented at the 2026 World Congress of Neuropsychopharmacology (CINP), scientists from National Taiwan University found that patients who improved after antidepressant treatment maintained a more balanced and functional microbial ecosystem, recovered beneficial metabolites, and displayed blood-based biological signals that aligned with these changes. http://www.bioworld.com/articles/732354 Tue, 30 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732354-cinp-2026-gut-microbiota-could-predict-antidepressant-response Illustration demonstrating gut-brain axis Gensci-134 (Genescience Pharmaceuticals Co. Ltd.) is a growth hormone receptor (GHR) agonist fusion protein that mimics the biological activity of growth hormone (GH), does not contain GH sequence and selectively activates human GHR for the treatment of GH deficiency (GHD). It is designed to enable once-every-4-week dosing. http://www.bioworld.com/articles/732314 Tue, 30 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732314-gensci-134-a-long-acting-growth-hormone-receptor-agonist-fusion-protein 3D illustration of human growth hormone molecules Human growth hormone. Genescience Pharmaceuticals Co. Ltd. has been developing an SLC6A19 inhibitor – Gensci-144 – for the potential treatment of chronic kidney disease (CKD). http://www.bioworld.com/articles/732211 Mon, 29 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732211-gensci-144-shows-renoprotection-in-ckd Illustration of diseased kidney The gut microbiota may be altered in people with depression as a result of treatment. These microorganisms reorganize differently in individuals who respond to therapy. In a multiomics study of antidepressant-naive patients presented at the 2026 World Congress of Neuropsychopharmacology (CINP), scientists from National Taiwan University found that patients who improved after antidepressant treatment maintained a more balanced and functional microbial ecosystem, recovered beneficial metabolites, and displayed blood-based biological signals that aligned with these changes. http://www.bioworld.com/articles/732204 Mon, 29 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732204-cinp-2026-gut-microbiota-could-predict-antidepressant-response Illustration demonstrating gut-brain axis At the BIO International Convention, nonprofit and for-profit companies discussed ways to fund orphan drug development, especially for ultra-rare diseases where development costs can be challenging. One of the simplest ways to keep costs down is to skip the discovery phase and use repurposed drugs or rescue shelved therapeutic assets as the Orphan Therapeutics Accelerator has done. The Cambridge, Mass.-based accelerator was founded in 2024 to rescue shelled therapeutic assets for ultrarare diseases. http://www.bioworld.com/articles/732173 Fri, 26 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732173-bio-2026-alternatives-to-funding-orphan-drugs Dollar sign inside vial, syringe Graves’ disease is a form of autoimmune hyperthyroidism where the main extrathyroidal feature is thyroid eye disease. Autoantibodies that stimulate thyroid-stimulating hormone receptor (TSHR) are released and orchestrate hormone production in the thyroid tissue and the orbit. Regeneron Pharmaceuticals Inc. has developed REGN-24493, a monoclonal antibody (MAb) that binds TSHR and blocks its activation by those autoantibodies. http://www.bioworld.com/articles/732191 Fri, 26 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732191-regeneron-reports-tshr-blocking-mab-for-graves-disease Thyroid anatomy Hypoparathyroidism is a rare disorder characterized by low levels of parathyroid hormone (PTH) that lead to low blood calcium levels and hyperphosphatemia. Septerna Inc. is developing a small-molecule PTH1 receptor (PTH1R) agonist, SEP-479, designed to replace PTH and normalize serum calcium. http://www.bioworld.com/articles/732190 Fri, 26 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732190-septerna-obtains-promising-preclinical-results-with-sep-479-for-hypoparathyroidism Photo of pipette with test tube Amyotrophic lateral sclerosis (ALS)-associated genes provide direct therapeutic targets and reveal pathways that can be used to develop treatments that counteract their harmful molecular effects. Because the underlying causes of most ALS cases remain unknown, identifying disease-associated variants is essential to uncover the mechanisms that drive the disease, as shown at the European Network to Cure ALS (ENCALS) meeting, held in Madrid from June 24 to 26, 2026. http://www.bioworld.com/articles/732185 Fri, 26 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732185-encals-2026-from-genetics-to-advancing-strategies-against-als Illustration for mutations in the DNA leading to brain diseases or neurodegenerative disorders The eighth version of the Prescription Drug User Fee Act (PDUFA) is up for renewal next year, but the bulk of the negotiations between the drug development industry and the U.S. FDA is largely complete. At the BIO International Convention, representatives from the industry and trade organization provided an overview of what was agreed upon during the 127 negotiating sessions that were needed to arrive at the commitment letter, which will be sent to Congress later in 2026. http://www.bioworld.com/articles/732158 Thu, 25 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732158-bio-2026-industry-negotiators-preview-pdufa-viii FDA logo on textured paper Credit: Araki Illustrations - stock.adobe.com Investigators from the Karl Landsteiner University aimed to evaluate the therapeutic potential of a novel CDK9 inhibitor/degrader, GT-02897, for the potential treatment of multiple myeloma. http://www.bioworld.com/articles/732134 Thu, 25 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732134-gt-02897-exerts-efficacy-in-preclinical-multiple-myeloma Researchers from Bristol Myers Squibb Co. (BMS) presented preclinical data on BMS-986453 (tunlucabtagene autoleucel), a dual-targeting BCMA×GPRC5D CAR T-cell therapy, in models of multiple myeloma. http://www.bioworld.com/articles/732132 Thu, 25 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732132-bmss-dual-car-t-cell-therapy-shows-efficacy-in-multiple-myeloma Lu-AG-22515, also known as velaprumig, is a recombinant fusion protein that targets CD40 ligand (CD40L) and human serum albumin (HSA), thereby blocking the binding between CD40 and CD40L, a known signaling pathway involved in several autoimmune diseases. Researchers at H. Lundbeck A/S evaluated the pharmacokinetic (PK) and pharmacodynamic (PD) profile of Lu-AG-22515 in cynomolgus monkeys, as well as the impact of its inhibitory effect in the preclinical setting of thyroid eye disease (TED). http://www.bioworld.com/articles/732119 Tue, 23 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732119-lundbecks-lu-ag-22515-shows-promise-in-thyroid-eye-disease Close up of eye and vision test Vascular-type bleeding disorder (BDVAS) is a rare, autosomal dominant disorder mainly caused by impaired vascular integrity. http://www.bioworld.com/articles/732118 Tue, 23 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732118-case-report-links-emapold1-em-variant-to-vascular-type-bleeding-disorder Illustration of red and white blood cells in an artery Acromegaly is a rare endocrine condition caused by hypersecretion of human growth hormone (GH) that results in elevated circulating levels of GH and insulin-like growth factor-1 (IGF-1). Opko Biologics Ltd. has presented data on the development and characterization of OPKO-8801001, a long-acting human GH receptor (GHR) antagonist with prolonged systemic exposure and pharmacological activity designed for subcutaneous administration. http://www.bioworld.com/articles/732113 Tue, 23 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732113-opko-biologics-characterizes-new-compound-for-acromegaly Pipet, test tubes, chemical structures Europe must focus on prevention and innovation to ensure its health care systems are sustainable in the long term, said Olivér Várhelyi, European commissioner for health and animal welfare. With an aging population, rising chronic diseases, a stretched workforce and geopolitical tensions, the universal health coverage that Europeans enjoy is under threat, he told delegates at the HLTH Europe conference in Amsterdam last week. http://www.bioworld.com/articles/732049 Mon, 22 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732049-europes-health-system-under-threat-prevention-and-innovation-key Female healthcare professional holding health icon Researchers at Theramir Ltd. presented preclinical proof-of-concept data for a new extracellular vesicle (EV) platform engineered to deliver miR-885-5p (EVmiR885-5p), targeting leukocyte-specific protein-1 (LCP-1)-mediated cytoskeletal remodeling in triple-negative breast cancer (TNBC) models. http://www.bioworld.com/articles/732097 Mon, 22 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732097-evmir-885-5p-limits-tnbc-spread-in-preclinical-models Illustration of triple-negative breast cancer cells The alternative pathway (AP) of the complement system is crucial for innate immunity and its dysregulation may lead to several diseases, including paroxysmal nocturnal hemoglobinuria (PNH). Alnylam Pharmaceuticals Inc. and Regeneron Pharmaceuticals Inc. recently presented data on ALN-CFB, a siRNA targeting hepatic complement factor B (CFB) that inhibits the production of CFB in the liver. http://www.bioworld.com/articles/732096 Mon, 22 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732096-preclinical-data-on-aln-cfb-for-paroxysmal-nocturnal-hemoglobinuria-presented Photo of pipette with test tube Light chain multiple myeloma (LCMM) is a cancer driven by malignant plasma cells that produce excessive pathogenic free light chains (FLCs) that may cause kidney dysfunction and form amyloid deposits in key organs, thus leading to poor outcomes. Ab Studio Inc.’s CATA-001 is a bispecific antibody targeting both CD38 and aggregated light chains (ALs) designed to deplete CD38+ plasma cells and clear both circulating and tissue-deposited pathogenic FLC aggregates for the treatment of LCMM and AL amyloidosis. http://www.bioworld.com/articles/732095 Mon, 22 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732095-cata-001-yields-promising-results-for-light-chain-multiple-myeloma Red and blue bispecific antibodies