https://www.bioworld.com/rss en-us The NIH Accelerating Medicines Partnership Program for Alzheimer’s disease (AD) has named tyrosyl-tRNA synthetase (TyrRS, YARS1) as a therapeutic target in AD. Functional Longevity Labs Inc. has developed FLL-001, a potential first-in-class small-molecule TyrRS modulator designed to restore TyrRS levels and rescue its triad in post-mitotic neurons for the treatment of AD. http://www.bioworld.com/articles/732710 Fri, 17 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732710-tyrrs-modulator-exerts-neuroprotection-in-ad-models Man piecing together a puzzle Researchers from the University of Strasbourg and the Centre National de la Recherche Scientifique (CNRS) have presented data on LIT-002, a nonpeptide oxytocin receptor (OXTR) agonist, for the potential treatment of autism and neuropathic pain. http://www.bioworld.com/articles/732709 Fri, 17 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732709-lit-002-is-strong-candidate-for-autism-and-neuropathic-pain Neuropathy illustrated in legs and feet Pathological tau accumulation is a key driver of neurodegeneration in Alzheimer’s disease (AD) and other tauopathies such as progressive supranuclear palsy. Researchers at Alnylam Pharmaceuticals and Regeneron Pharmaceuticals Inc. presented preclinical efficacy data on ALN-5288 in tauopathy models. http://www.bioworld.com/articles/732704 Fri, 17 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732704-aln-5288-lowers-tau-levels-in-preclinical-models Signs of recovery are emerging in the biotechnology investment sector, although investors across Asia say that capital is flowing selectively to companies that can demonstrate clinical differentiation, strong management teams and a clear path to commercialization. http://www.bioworld.com/articles/732692 Thu, 16 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732692-capital-remains-selective-in-barbell-biotech-market International currency symbols Reduced endocannabinoid signaling has been linked to neuronal hyperexcitability in Alzheimer’s disease (AD), suggesting that dual inhibition of fatty acid amide hydrolase (FAAH) and monoacylglycerol lipase (MAGL) may restore endocannabinoid tone and alleviate hyperexcitability-driven symptoms, including agitation, a common neuropsychiatric manifestation of AD. http://www.bioworld.com/articles/732678 Thu, 16 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732678-dual-faah-magl-inhibition-targets-alzheimers-disease-hyperexcitability Elderly hands holding broken brain structure Pyroglutamate-modified amyloid-β (pE3-Aβ) is a highly pathogenic Aβ species that accumulates within amyloid plaques and contributes to aggregation, neuroinflammation and neuronal dysfunction in Alzheimer’s disease (AD). Researchers from Sound Biologics (Qilu Puget Sound Biotherapeutic Corp.) described the preclinical profile of PSB-229, a novel brain-shuttled anti-pE3-Aβ antibody in models of AD. http://www.bioworld.com/articles/732677 Thu, 16 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732677-psb-229-reduces-plaque-burden-in-alzheimers-models Brush scrubbing brain figurine with soap Excessive fibrinolysis is a major driver of bleeding across multiple clinical settings, including heavy menstrual bleeding and other hemorrhagic disorders. Researchers from Hemab ApS presented preclinical data on HMB-003, a long-acting plasmin inhibitor that directly targets the enzyme’s active site to achieve sustained antifibrinolytic activity. http://www.bioworld.com/articles/732673 Thu, 16 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732673-hmb-003-outperforms-tranexamic-acid-in-fibrinolysis-models FTO is an α-ketoglutarate-dependent RNA demethylase that regulates N6-methyladenosine (m6A), one of the most abundant epitranscriptomic modifications in mammalian mRNA. Emerging evidence suggests that aberrant m6A signaling may contribute to neuronal dysfunction and neurodegeneration. Researchers at the University of Barcelona and collaborators described the discovery and preclinical characterization of a new series of FTO inhibitors designed to target neurodegenerative disease by modulating m6A RNA demethylation. N6-methyladenosine (m6A), one of the most abundant epitranscriptomic modifications in mammalian mRNA. Emerging evidence suggests that aberrant m6A signaling may contribute to neuronal dysfunction and neurodegeneration. Researchers at the University of Barcelona and collaborators described the discovery and preclinical characterization of a new series of FTO inhibitors designed to target neurodegenerative disease by modulating m6A RNA demethylation.]]> http://www.bioworld.com/articles/732649 Wed, 15 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732649-new-fto-inhibitor-shows-neuroprotective-effects Illustration of head with maze that is missing parts At the Alzheimer’s Association International Conference, researchers from Voyager Therapeutics Inc. presented preclinical efficacy data for VY-1706, a blood-brain barrier-penetrant AAV9 gene therapy designed to reduce tau levels in models of Alzheimer’s disease (AD). http://www.bioworld.com/articles/732646 Wed, 15 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732646-voyagers-gene-therapy-reduces-tau-levels-in-ad-models Illustration of human brain and dna The prophylaxis of bleeding disorders usually requires frequent intravenous infusions that result in excessive treatment burden and residual bleeding risk. TGM-148 is a siRNA developed by Tangram Therapeutics plc designed to repress the hepatocyte expression of a gene target to rebalance hemostasis, and has shown efficacy in murine models of hemophilia and von Willebrand disease. http://www.bioworld.com/articles/732643 Wed, 15 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732643-preclinical-data-support-continued-development-of-tgm-148-for-bleeding-disorders Tuesday brought what was arguably the most anticipated presentation of the 2026 Alzheimer’s Association International Conference when Catherine Mummery, head of novel therapeutics at University College London’s Dementia Research Center, presented data from the phase II Celia trial of tau-lowering antisense oligonucleotide diranersen (BIIB-080, Biogen Inc.). Based on both clinical and biomarker data, “Celia establishes a proof of concept,” Mummery said, that reducing tau may slow the progression of Alzheimer’s disease. http://www.bioworld.com/articles/732664 Tue, 14 Jul 2026 11:00:00 -0400 https://www.bioworld.com/articles/732664-celia-trial-data-strong-analysts-skeptical Senior medical exam Aggregated α-synuclein (α-Syn) is encoded by the SNCA gene and is one of the key pathological proteins linked to Parkinson’s disease. However, it is still not clear whether or not α-Syn originating in the periphery has a role in the pathology in the brain. SNCA gene and is one of the key pathological proteins linked to Parkinson’s disease. However, it is still not clear whether or not α-Syn originating in the periphery has a role in the pathology in the brain.]]> http://www.bioworld.com/articles/732630 Tue, 14 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732630-new-model-explores-peripheral-syns-impact-on-brain Neurology illustration Human cytomegalovirus (CMV) is a clinical challenge in infants and immunocompromised individuals, since no licensed vaccine exists. Vaccine development is complex due to several factors, including viral latency, genomic complexity and immune evasion, among others. Evaxion A/S recently presented data on its proprietary AI-immunology platform used to create the EVX-V1 vaccine for CMV infection. http://www.bioworld.com/articles/732627 Tue, 14 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732627-preclinical-data-support-evaxions-platform-behind-cmv-vaccine-candidate Cytomegalovirus in a human cell Anorexia nervosa (AN) is a complex and severe eating disorder that has long been considered a psychiatric condition driven by distorted body image and maladaptive eating behaviors. No effective therapy is currently available. At a symposium held during the recent FENS Forum in Barcelona, several leading researchers with decades of experience in AN presented findings that further strengthen the growing body of evidence supporting the disorder’s metabolic component. http://www.bioworld.com/articles/732624 Tue, 14 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732624-rethinking-anorexia-a-disorder-of-mind-and-metabolism Photo of scale with woman sitting on floor holding head and arms on knees Researchers from Connecta Therapeutics SL and the Centre for Genomic Regulation (Barcelona Institute of Science and Technology) recently presented preclinical results on CTH-120, a potential first-in-class allosteric modulator of tropomyosin receptor kinase B (TrkB) designed to enhance neuroplasticity and restore neuronal function in fragile X syndrome. http://www.bioworld.com/articles/732567 Mon, 13 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732567-connectas-trkb-allosteric-modulator-shows-promise-in-fragile-x-model Brain and DNA Researchers from United Arab Emirates University presented the preclinical characterization of ST-2657, a dual G9a/H3R modulator, in a preclinical model of autism. http://www.bioworld.com/articles/732566 Mon, 13 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732566-dual-g9a-h3r-modulator-exhibits-efficacy-in-autism-model Colorful silhouette amidst grayscale silhouettes The Alzheimer’s Association International Conference (AAIC) is the world’s biggest dementia conference. And at the AAIC 2026 meeting, there is big buzz around tau. Sunday’s plenary speaker Ryan Watts, CEO of Denali Therapeutics Inc., highlighted tau-lowering agents as being among the most exciting themes of the conference. “At this conference, we’re going to see additional clinical data that may validate [tau as] a second target in Alzheimer’s disease, amyloid being the first,” he told the audience. http://www.bioworld.com/articles/732563 Mon, 13 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732563-aaic-2026-age-of-amyloid-is-being-joined-by-time-of-tau AI-generated illustration of a neuron with tau protein aggregation Researchers from Flavii Therapeutics SL and collaborators presented preclinical data on FLAV-27, a G9a inhibitor designed to target G9a-mediated epigenetic dysregulation. http://www.bioworld.com/articles/732548 Fri, 10 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732548-flav-27-improves-asd-like-behaviors-in-btbr-mice 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