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✨ Today's Pharma Daily Digest brings you essential insights driving the future of #neuroscience treatment breakthroughs. Dive into the latest, must-know developments shaping the industry.   🔺 ZILBRYSQ® (zilucoplan) Injection Secures South Korea Approval, Offering New Hope for Myasthenia Gravis Patients 🔺 Trace Neuroscience, a newly launched biotech company originating from Maze Therapeutics' pipeline, has raised $101 million  to develop innovative treatments targeting UNC13A, aiming to revolutionize care for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions. 🔻 Neurogene Inc.'s Phase I/II Rett syndrome trial faced a setback as a patient treated with the higher 3E15-vg dose of NGN-401 died from systemic hyperinflammatory syndrome, a rare but severe side effect of adeno-associated virus (AAV) gene therapies. Despite this tragedy, the FDA has approved the continuation of the trial at the safer 1E15-vg dose ----- 💡 Sign up for our Daily Digest to stay updated on the pharma and biotech advancements shaping tomorrow’s healthcare breakthroughs. Don’t miss the insights that could impact your next strategic move! #PharmaDailyDigest #Pharmaceuticals #Biotech #CompetitiveIntelligence #AtacanaGroup #neurology #neuroscience #neurogene #traceneuroscience #Myasthenia

Impact of CRISPR in drug discovery "CRISPR-based screens of cell lines, and phenotypic readouts such as cell growth, can facilitate the discovery of genetic interactions that could be therapeutic targets. Machine learning of these datasets and other information can be used to model drug effects on tumor microenvironments and better predict effects on patients." This technology can be applied to any disease, particularly for those with clear cellular phenotypes, such as lysosomal storage diseases. This Perspective article, focused on cancer drug discovery, was published in Science: https://lnkd.in/eR55ENje. #genetics #genomics #precisionmedicine #genomicmedicine #cancer #brain #neurology #neurodegeneration #neuroscience #neuroinflammation #lysosomalstoragedisease #drugdiscovery #drugdevelopment #ai #omics #biotechnology #pharma #innovation #research #science #sciencecommunication

Modeling Lewy body disease with SNCA triplication iPSC-derived cortical organoids and identifying therapeutic drugs Researchers modeled Lewy body disease (LBD) using human-induced pluripotent stem cell-derived cortical organoids from patients with SNCA gene triplication. They identified synaptic and mitochondrial dysfunction in excitatory neurons with high SNCA expression. A drug screen revealed that entacapone, tolcapone, phenazopyridine hydrochloride, and zalcitabine, all FDA-approved compounds, inhibited α-SYN aggregation and alleviated mitochondrial dysfunction, suggesting potential therapeutic targets for LBD. The article was published in Science Advances: https://lnkd.in/eRSY2d9V. #genetics #genomics #precisionmedicine #genomicmedicine #brain #neurology #neuroscience #neurodegeneration #parkinson #lbd #metabolism #physiology #cardiovasculardisease #mitochondia #drugrepurposing #neurons #therapeutics #biotechnology #innovation #research #science #sciencecommunication

Millions of people worldwide are affected by neurodegenerative disorders, primarily characterized by neuronal dysfunction and loss. Still, there are no effective treatments available and deeper knowledge about the early processes triggering the disease cascade is fundamental for development of therapeutics for these diseases. At Cellectricon, we help our clients to gain insight about the early processes triggering the disease cascade by applying in vitro models to investigate how different cell types, genes and cellular mechanisms are involved in disease progression. Our ND service module is based on complex CNS disease models where aspects of neurodegeneration, such as protein aggregation and cellular toxicity, are replicated in vitro. Gene silencing or overexpression technologies can be used to investigate which genes and pathways that directly or indirectly influence the pathogenesis of the disease process. Reach out to learn more about our services on: https://lnkd.in/edQ-5E88 #CRO #neuroscience #drugdiscovery #targetdiscovery #leaddiscovery #neurobiology #invitro #research #neurodegeneration #parkinsons #alzheimers

Check out our new study: "Single-Cell Multi-Omics Map of Cell Type-Specific Mechanistic Drivers of Multiple Sclerosis Lesions." We identified a unique genetic signature in oligodendrocytes and spatially distinct metabolic changes in astrocytes at chronic active lesions in patients with progressive multiple sclerosis. Read more here: https://lnkd.in/eXfF2vXp #MultipleSclerosis #Neuroscience #SingleCellResearch

🧠 Exciting Breakthrough in Multiple Sclerosis Research- A New Mechanism to Regenerate Myelin? This groundbreaking discovery, published in Cell, highlights the potential of a novel protein function inhibitor, ESI1, to regenerate myelin and restore neurological function. 🔬 Key Insights: ESI1 targets gene silencing in oligodendrocytes, boosting myelin production. Successful preclinical tests in mice and human brain cells suggest potential for human trials! This approach could revolutionize MS treatment and extend to other neurodegenerative conditions. 💡 Implications for the future: Potential to reverse nerve damage in MS and similar disorders. Recovery from brain and spinal injuries. Opens possibilities for combating age-related cognitive decline. #MSresearch #Neuroscience #MedicalBreakthroughs #mutiplesclerosis #myelin #neurodegnerative #regenerativemedicine

New Neuroscience Article! Huntington’s disease (HD) is an inherited neurodegenerative disease caused by a CAG extension in exon 1 of the huntingtin gene. An extended polyglutamine tract in the huntingtin protein is developed due to the expanded alleles, resulting in intracellular signalling defects.Antisense Oligonucleotide (ASO) gene therapy is currently being pioneered to treat HD………… Read the rest of the article: https://lnkd.in/gma_icsT Written by: Maria K Neuroscience News British Neuroscience Association (BNA) Neuroscience, Surgery and Trauma Centre #neuroscience #huntington #huntingtondisease #genetherapy #disease

Deletion of GBA in neurons, but not microglia, causes neurodegeneration in a Gaucher mouse model: implications for disease therapeutics Gaucher disease (GD), caused by homozygous GBA1 gene mutations, is characterized by microgliosis, astrogliosis, and neurodegeneration, while GBA1 carriers present an increased risk of developing Parkinson's disease. Researchers created cell-type-specific GBA KO mice to understand the contribution of microglia and neurons to disease pathogenesis. Microglia-specific GBA KO mice showed no disease, while neuron-specific GBA KO mice exhibited severe neurodegeneration, weight loss, and shortened lifespan. These findings suggest that neuron-specific GBA deficiency is primarily responsible for neuropathic GD symptoms, and therefore, this cell type should be targeted in therapeutic interventions. The article was published in JCI Insights: https://lnkd.in/eqrBb-T8. We obtained similar results for Niemann-Pick C several years ago: https://lnkd.in/eVstt4yu, https://lnkd.in/eXCekJMd. #genetics #genomics #precisionmedicine #genomicmedicine #brain #neurology #neurodegeneration #neuroscience #gaucher #raredisease #lysosomalstoragedisorders #biomarkers #therapeutics #drugdevelopment #biotechnology #innovation #research #science #sciencecommunication

 Gold-plated neurons! ⭐️ Okay, no, not exactly, but how about gold nanoparticles as an alternative to optogenetics? Researchers at the University of Chicago have devised a method to stimulate neurons using gold nanoparticles (AuNPs) conjugated with antibodies. This approach is a real alternative to optogenetics as it allows you to induce neuron activation without genetic modification.  The team used antibodies, like our Anti-Rat TRPV1 (extracellular) Antibody (ACC-029) and Anti-P2X3 Receptor (extracellular) Antibody (APR-026) and conjugated them to AuNPs to target specific membrane proteins and localize photostimulation. They manually removed BSA and sodium azide from their antibody solutions to help with the conjugation. What they could have done was get the carrier-free versions from us at the start and save all that time and effort of doing it themselves! This work has significant potential in both research and therapeutic applications, especially when it comes to investigating neurological disorders and potentially restoring vision in degenerative eye diseases. Get all the details published in Neuron 👇 https://lnkd.in/eCJCJ9xp Cell Press University of Chicago University of Illinois Chicago Stephen Kent Bobo Dang David Pepperberg #nanoparticles #neuroscience #optogenetics #antibodies

What is microRNA? These tiny molecules have revolutionised our understanding of gene regulation and hold immense potential for transforming medicine. From cancer to heart disease, microRNA-based therapies are the future of personalised healthcare. Swipe through to learn about the Nobel-winning discovery that’s changing the game for human health! #microRNA #GeneTherapy #NobelPrize2024 #MedicalBreakthrough #HealthcareInnovation #ScienceExplained #PositiveNews #Cancer #HeartHealth #Neuroscience [microRNA, Nobel Prize 2024, Nobel-winning discovery, medical science, Neuroscience, Victor Ambros, Gary Ruvkun]