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Explore our in-depth analysis of the #FDA's regulatory push and #China's accelerating progress in the #global shift 👉 https://lnkd.in/eS3F4akG #OrganOnAChip #DrugDevelopment #Biotech #Policy #ChinaInnovation #VCBeat

When Brett P. Monia, PhD, first joined Ionis more than 35 years ago, he was part of a small group of scientists chasing what many considered an impossible dream: creating an entirely new way to design medicines with genetic precision.    Today, under his leadership and through the dedication of more than more than 1,200 Ions, Ionis has become a fully integrated biotech, accelerating the delivery of RNA-targeted medicines from discovery through commercialization with the goal of enabling better futures for people living with serious diseases.    And this is just the beginning. Discover how Ionis is shaping the future of RNA-targeted medicines: https://lnkd.in/eQHKKZ74   #RNAmedicines #RNATherapeutics #PatientImpact #Ionis 

When “Absolutely Impossible” Becomes Discovery Back around 2011–2012, after a seminar by my student Jason Baik, a senior colleague declared that it was “absolutely impossible” for clofazimine—a single small molecule—to phase-separate and crystallize inside macrophages as the drug was orally administered to live animals. That phrase—absolutely impossible—has echoed in my mind ever since. Yet over the next decade, my lab at the University of Michigan demonstrated exactly that: clofazimine forms intracellular drug inclusions within macrophages, giving rise to a previously unrecognized class of cells I later termed “Xenobiotic Sequestering Cells” (XSCs). These cells stabilize crystalline inclusions within expanded lysosomes, remaining resistant to lysosome-induced apoptosis—a biological adaptation that allows the body to sequester and tolerate persistent foreign compounds. This mechanism of macrophage-stabilized phase separation of xenobiotics was not supposed to exist. My research group published extensively about how clofazimine could be used as a probe of macrophage differentiation into xenobiotic sequestering (inclusion body-forming) cells, and to explore their potential role in the “Absolutely Impossible” pharmacokinetics of other molecules exhibiting extremely long half-life. Even today, leading scientists such as Nobel Laureate Drew Weissman seem to deny that such a phenomenon could occur, stating it is “Absolutely Impossible” for stabilized mRNA LNPs to persist in the body for as long as two years, after an intramuscular injection. But science progresses when imagination inspires scientists to ask the right questions. What began as a “heretical” observation evolved into a new paradigm, with our studies linking intracellular phase transitions to long-term drug distribution, efficacy and toxicity. It’s a story of persistence, skepticism, and independence. So, as I now decide to step away from my faculty role and into a new creative phase of my life, I do so with deep gratitude and conviction. Not as an artist, but as a free thinker who chooses truth over hype, and discovery over conformity. There are many things we still do not understand about how the body responds to xenobiotics and drugs. But one thing is certain: Macrophages can sequester and crystallize xenobiotics in a way that challenges our assumptions about what is Absolutely Impossible, until we shatter them. — Gus Rosania, Ph.D. Professor (soon to be “Retired”) of Pharmaceutical Sciences University of Michigan

💊 XORTX Therapeutics Inc.: Expands Kidney Disease Pipeline with Strategic Acquisition from Vectus Biosystems XORTX Therapeutics Inc. (Nasdaq: XRTX) has entered into a binding term sheet to acquire Vectus Biosystems’ Renal Anti-Fibrotic Therapeutic Program, featuring VB4-P5, a novel small molecule with potential to treat both rare and large-market kidney diseases. 📄 Learn more about the acquisition and XORTX’s kidney disease pipeline: https://lnkd.in/eAz5xqME PharmaVoice Genetic Engineering & Biotechnology News Biotechnology #XRTX #BiotechStocks #KidneyDisease #PharmaNews #HealthcareInnovation #Investing #CKD #RareDiseases

Spotlight on Cytion’s Human Primary Cells 🧬 At 2BScientific GmbH, we’re proud to partner with Cytion, a trusted cell bank offering a diverse portfolio of human primary cells including HUVECs, gingival fibroblasts (hGF), dental follicle stem cells (hDFSC), and more. These cell types are particularly useful for studies in: • Vascular biology and angiogenesis (HUVECs) • Wound healing and tissue repair (hGF) • Oral biology and regeneration (hDFSC, gingival fibroblasts) • Drug development, toxicology, and disease modeling Find out more here: https://lnkd.in/eb8dKyQH #PrimaryCells #CellBiology #HumanModels #TranslationalResearch #Cytion #LifeSciences #InVitroModels

I’m currently working on a project aimed at enhancing transdermal delivery of Mupirocin, an effective topical antibiotic whose clinical impact is often limited by permeation challenges and emerging resistance. The study combines experimental approaches with molecular dynamics (MD) simulations, which serve as a powerful predictive tool providing atomistic insights into drug–lipid interactions. MD simulations reveal binding behavior, structural dynamics, and lipid preferences, complementing experiments and guiding the rational design of more effective and stable topical drug delivery systems. This ongoing work aims to develop a biocompatible and efficient nanocarrier system for topical antibiotics, offering insights that could advance drug delivery strategies and therapeutic performance. #MolecularDynamics #Liposomes #PolymericNanofilm #DrugDelivery #Nanomedicine #AntimicrobialResearch #SkinTherapy

🌐 From frameworks to foresight: today, the CGT materials community came together to set new benchmarks. 🔬 Wrapping up Day 1 at the 2nd Raw Materials for Cell & Gene Therapy Summit — what a start! Honored to take part as a speaker at this year’s event in Boston, a meeting that’s fast becoming the definitive forum for how our field defines, qualifies, and manages the raw materials that make advanced therapies possible. Day 1 was filled with powerful insights and forward-thinking dialogue focused on turning conversation into implementation. From Dawn Landis-Henke (Standards Coordinating Body) setting the tone on evolving standards, to Basak Clements, Grace Perry (Roche), Thomas Liu (Adaptimmune), Naeem (Nick) Sarwar (Vertex Pharmaceuticals), Benjamin Espen (Avobis Bio), Randal Alfano Randall A. (Sartorius), Charles Burt (Abeona Therapeutics), and Andreas M. Beckhaus & David Cady (ProDeMaCon) sharing practical strategies across qualification, supply resilience, and risk control, the message was clear: Materials matter just as much as molecules. In my session, I discussed Quality by Design (QbD) and risk-based raw material qualification, emphasizing that the next step is predictive quality, where material intelligence and data analytics drive proactive control and consistency. Here are my 7 takeaways from Day 1: 1️⃣ QbD is evolving: from reactive to predictive, transforming material data into actionable insight. 2️⃣ Defining “GMP raw materials” remains a global challenge: harmonization across pharmacopeias and regulators is essential. 3️⃣ Supplier partnerships are strategic assets: transparency, co-development, and risk-based collaboration are key. 4️⃣ Risk is dynamic, not static: frameworks like USP <1043> and ISO 20399 must evolve alongside process maturity. 5️⃣ Characterization connects science and safety: linking CMAs to CQAs ensures consistent, patient-centric outcomes. 6️⃣ Documentation builds trust: CoA, CoO, E&L, and full traceability underpin true GMP readiness. 7️⃣ Collaboration defines the future: progress depends on joint innovation among industry, suppliers, regulators, and standards bodies. The energy in the room was exceptional; a shared commitment to build the future infrastructure of CGT manufacturing through science, quality, and collaboration. Grateful to contribute alongside such outstanding peers and looking forward to Day 2 of inspiring dialogue and discovery. #CellTherapy #GeneTherapy #AdvancedTherapies #RawMaterials #Biomanufacturing #QbD #CGT #RegulatoryScience #Leadership #Innovation #Standards

Compared to ex vivo and in vivo glaucoma models, 𝒊𝒏 𝒗𝒊𝒕𝒓𝒐 𝐜𝐞𝐥𝐥 𝐠𝐥𝐚𝐮𝐜𝐨𝐦𝐚 𝐦𝐨𝐝𝐞𝐥𝐬 offer several distinct advantages. They are rapidly constructed, relatively inexpensive, reproducible and at the same time provide valuable mechanistic insights into glaucoma disease. 𝐀𝐜𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬 offers a wide range of customized 2D or 3D cell models: • Retinal ganglion cells - primary cells damaged in glaucoma • Trabecular meshwork cells - play a central role in regulating intraocular pressure • Astrocyte - key mediators of inflammation in glaucoma • Microglia - key mediators of inflammation in glaucoma Leveraging our custom cell models, we also provide comprehensive services to support: • Cell physiology study • Basic research • Drug discovery & development • Toxicology study • Permeability study From model development to experiments & data analysis, 𝐀𝐜𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬 offers comprehensive support throughout your glaucoma project. Learn more: https://lnkd.in/drVzPh9T #AceTherapeutics #AcceleratingDiscovery #Glaucoma #GlaucomaModels #CellModels #GlaucomaResearch

Alterity Therapeutics Presents Positive Data from ATH434 Phase 2 Trial at the 2025 International Congress of Parkinson’s Disease and Movement Disorders #MDSCongress #MovementDisorders #MultipleSystemAtrophy #biotech #ClinicalTrials $ATHE $ATH Read the release here - https://lnkd.in/eq44GiSp

SFBN Feed: Robotic pill maker Rani teams up with Chugai in deal worth up to $1.08B https://lnkd.in/dVPvyq6K Rani Therapeutics is pairing its oral delivery system with Chugai’s experimental rare disease antibody as part of a deal worth up to $1.08 billion. San Jose, CA-based Rani also disclosed a … Click here to [...] #BayArea #SanFrancisco #Biotech #Lifescience #News