World Pharmaceutical Frontiers’ Post

Can a single gene injection resurrect a failing heart – sounds like science fiction, isn’t it? Except, it’s not. It is a breakthrough in medical science unfolding right now. Researchers at the University of Utah's Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) have made groundbreaking advancements in treating heart failure through innovative gene therapy. This novel approach, focusing on the cardiac bridging integrator 1 (cBIN1) gene, has demonstrated unprecedented efficacy in reversing heart failure in a large animal model. The study, published in NPJ Regenerative Medicine, utilized a porcine model of non-ischemic heart failure with reduced ejection fraction (HFrEF). The therapy involves administering a harmless virus vector carrying the cBIN1 gene, which is delivered to cardiac cells via bloodstream injection. Key findings of the study: 1. Functional Improvement: The gene therapy resulted in a remarkable 30% improvement in heart function, significantly surpassing previous therapeutic attempts that typically achieved only 5-10% improvement. 2. Survival Rate: All treated subjects survived the entire six-month study duration, a notable outcome given the typically poor prognosis of untreated heart failure in this model. 3. Reverse Remodeling: The treatment induced reverse remodeling of the heart, with treated hearts exhibiting less dilation and thinning, more closely resembling non-failing cardiac morphology. 4. Molecular Mechanism: cBIN1 functions as a centralized signaling hub, regulating multiple downstream proteins and organizing critical heart cell functions. This scaffolding role of cBIN1 is hypothesized to be key to its therapeutic efficacy. 5. Microscopic Improvements: The therapy led to better-organized heart cells and proteins at the microscopic level, suggesting a comprehensive restoration of cardiac ultrastructure. The researchers, led by Dr. Robin Shaw and Dr. TingTing Hong, posit that cBIN1's role as a master regulator of heart cell architecture could introduce a new paradigm in heart failure treatment, directly targeting heart muscle function. While these results are encouraging, human clinical trials are still needed to determine the therapy's effectiveness and safety in people. In collaboration with TikkunLev Therapeutics, the team is adapting the therapy for human application, with plans to seek FDA approval for clinical trials in fall 2025. This research represents a potential paradigm shift in cardiac medicine, as the ability to halt the progression and potentially reverse cardiac damage could revolutionize treatment strategies and significantly improve patient outcomes. #MedicalInnovation #CardiacResearch #GeneTherapy https://lnkd.in/ewkQeSM7

Exciting Development in Back Pain Treatment! A recent study in Biomaterials presents a breakthrough non-viral gene therapy for discogenic back pain (DBP). Researchers have successfully used engineered extracellular vesicles (eEVs) to deliver the transcription factor FOXF1 to degenerative intervertebral discs (IVDs) in animal models. Key Highlights: Innovative Approach: FOXF1-loaded eEVs target and repair damaged IVDs, addressing chronic low back pain. Promising Results: The therapy restored disc structure and function, enhanced tissue hydration, and reduced pain behaviors in mice over 12 weeks. Future Potential: This method offers a non-addictive, less invasive alternative to current treatments, showing significant promise for human application. This groundbreaking therapy could revolutionize how we treat DBP, potentially improving the quality of life for millions suffering from chronic back pain. https://lnkd.in/ejCuFfWX Don't let searching for a new #PCRWorkstation for your #GeneTherapy #Research be a pain in your back! Drop me a line! I'm Happy to help! [email protected] or 919-754-3730 #GeneTherapy #BackPain #Biomaterials #MedicalResearch #InnovationsInHealthcare #PCR #PCRWorkstation #AirCleanSystems #Research #Innovation #Therapy #Gene #DNA #RNA

Cell therapy and gene therapy (CGT) are some of the fastest growing fields in modern healthcare and could fundamentally change medicine as we know it today

What is Duchenne Muscular Dystrophy(DMD)? DMD is a rare and devastating neuromuscular disorder caused by mutations in the dystrophin gene. DMD leads to progressive muscle weakness that worsens over time, ultimately affecting a person's ability to walk, breathe, and perform everyday tasks. It is an X-linked disorder, meaning it primarily affects males. It is fatal and occurs in approximately 1 in every 3500-5000 male births worldwide. In a recent breakthrough, the FDA has expanded approval for Elevidys, the first gene therapy designed to treat DMD. Elevidys works by introducing an engineered gene that codes for a shortened version of dystrophin. Click on the link below to learn more! https://lnkd.in/grFNRYJp

🧬 40 𝐚𝐩𝐩𝐫𝐨𝐯𝐞𝐝 𝐜𝐞𝐥𝐥 𝐚𝐧𝐝 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬! The year is almost over, but FDA approvals in the CGT space don’t stop. Last week, Autolus Therapeutics received approval for Aucatzyl – a cell therapy for relapsed/refractory B-cell acute lymphoblastic leukaemia. Today, PTC Therapeutics, Inc. announced the approval of Kebilidi – a gene therapy for the ultra-rare disease AADC deficiency. This is also the first gene therapy administered directly to the brain. With these two approvals, CGT hits a beautiful number 40. Here are the therapeutic areas: ⚆ haematology (21) ⚆ oncology (6) ⚆ neurology (5) ⚆ dermatology (3) ⚆ immunodeficiency (1) ⚆ endocrinology (1) ⚆ ophthalmology (1) ⚆ musculoskeletal (1) ⚆ aesthetic (1) 𝘞𝘪𝘭𝘭 𝘸𝘦 𝘴𝘦𝘦 𝘮𝘰𝘳𝘦 𝘍𝘋𝘈 𝘢𝘱𝘱𝘳𝘰𝘷𝘢𝘭𝘴 𝘧𝘰𝘳 𝘊𝘎𝘛 𝘵𝘩𝘪𝘴 𝘺𝘦𝘢𝘳? #approval #fda #genetherapy #pharma #biotech #drjojo *Certain therapies might fall under more than one category. ---------- Hi! I am Joanna, and my friends call me Dr Jojo 🌸 𝐈 𝐡𝐞𝐥𝐩 𝐬𝐭𝐚𝐫𝐭𝐮𝐩𝐬 𝐚𝐧𝐝 𝐞𝐧𝐭𝐫𝐞𝐩𝐫𝐞𝐧𝐞𝐮𝐫𝐬 𝐦𝐚𝐫𝐤𝐞𝐭 𝐭𝐡𝐞𝐢𝐫 𝐯𝐚𝐥𝐮𝐞 𝐚𝐧𝐝 𝐢𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧 ♻️ Share this if you find it interesting 🔔 Follow for insights you won't want to miss 📩 DM me to increase your visibility, grow your business, and generate leads

🚀 𝗔𝗱𝘃𝗮𝗻𝗰𝗶𝗻𝗴 𝗶𝗣𝗦𝗖-𝗗𝗲𝗿𝗶𝘃𝗲𝗱 𝗖𝗲𝗹𝗹 𝗧𝗵𝗲𝗿𝗮𝗽𝗶𝗲𝘀: 𝗞𝗲𝘆 𝗜𝗻𝘀𝗶𝗴𝗵𝘁𝘀 𝗳𝗿𝗼𝗺 𝘁𝗵𝗲 𝗟𝗮𝘁𝗲𝘀𝘁 𝗜𝗻𝗱𝘂𝘀𝘁𝗿𝘆 𝗥𝗲𝘃𝗶𝗲𝘄! 🌿 The recent review by the JSRM-ISCT, International Society for Cell & Gene Therapy iPSC Committee delves into the critical 𝘤𝘩𝘢𝘭𝘭𝘦𝘯𝘨𝘦𝘴 𝘢𝘯𝘥 𝘰𝘱𝘱𝘰𝘳𝘵𝘶𝘯𝘪𝘵𝘪𝘦𝘴 𝘧𝘰𝘳 𝘪𝘗𝘚𝘊-𝘣𝘢𝘴𝘦𝘥 𝘵𝘩𝘦𝘳𝘢𝘱𝘪𝘦𝘴. Here are 3 take-home messages: 𝟭- 𝗛𝗮𝗿𝗺𝗼𝗻𝗶𝘇𝗶𝗻𝗴 𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗦𝘁𝗮𝗻𝗱𝗮𝗿𝗱𝘀 🌍: Navigating the diverse global regulatory frameworks is essential for ensuring the safe and efficient development of iPSC-derived therapies. Early engagement with regulatory bodies can help streamline product approval. 𝟮- 𝗙𝗼𝗰𝘂𝘀 𝗼𝗻 𝗤𝘂𝗮𝗹𝗶𝘁𝘆 𝗖𝗼𝗻𝘁𝗿𝗼𝗹 & 𝗖𝗼𝘀𝘁 𝗠𝗮𝗻𝗮𝗴𝗲𝗺𝗲𝗻𝘁 💡: The path to commercialization requires rigorous quality control and thoughtful cost-of-goods (COGs) strategies. Optimizing manufacturing processes through advanced automation can significantly reduce expenses, making therapies more accessible. 𝟯- 𝗜𝗻𝗻𝗼𝘃𝗮𝘁𝗶𝘃𝗲 𝗔𝗽𝗽𝗿𝗼𝗮𝗰𝗵𝗲𝘀 𝘁𝗼 𝗧𝘂𝗺𝗼𝗿𝗶𝗴𝗲𝗻𝗶𝗰𝗶𝘁𝘆 𝗥𝗶𝘀𝗸𝘀 🔬: Addressing the risks of genomic instability and tumorigenicity remains a major challenge. Collaborative efforts and new assay developments are key to ensuring the safety and efficacy of these transformative treatments. #CellTherapy #iPSC #RegenerativeMedicine #BiotechInnovation #QualityControl #GlobalHealth https://lnkd.in/eHXRz7e2 Stephen Sullivan, PhD, MBA, FRSM iPSirius Lindville Bio Veronica Falco Dominic Wall Cell Therapies Pty Ltd Nathan Smith

✔️ The number of Cell and Gene Therapies available to patients continues to grow - CTs are mostly used to treat blood disorders, such as leukemia, with disordered cells being replaced. Gene Therapies, on the other hand, are used to treat a wide range of diseases, from skin cancer to ocular disorders - and most recently, with Kebilidi, brain disorders. Kebilidi uses an AAV vector due to the unique ability of these <50 nm particles to cross the blood-brain barrier, taking their genetic load with them. 🔸 Vector development has been key to the CGT revolution, with the choice and strategic modification of the delivery system allowing focussed, effective targeting of high-cost cargos. For more info, please see here: https://lnkd.in/g9P3YNdv #AAV #LNP #GeneTherapy #Kebilidi #MalvernPanalytical

Gene therapy is revolutionizing modern medicine, but finding a safe and effective delivery vehicle remains a critical challenge. While liposomes offer significant promise over viral-based delivery methods, they struggle with limited delivery efficiency across various cell types. A key obstacle is our incomplete understanding of the cellular structures necessary for liposome uptake, particularly in migratory versus epithelial cells. In our latest research, we've discovered the cellular structures required for efficient liposome-based gene delivery using human pluripotent stem cells. This advancement could pave the way for more effective and safer gene therapy applications in the future. #GeneTherapy #BiomedicalResearch #StemCells #Liposomes #MedicalInnovation #NIGMS #NIH #NEI Basic and Translational Research, Dept of Ophthalmology, IUSM https://lnkd.in/gvCfnAwM

🚨 New Market Report 🚨 The Retinitis Pigmentosa Treatment Market is poised for significant growth from 2024-2028, driven by the increasing prevalence of the condition and advancements in treatment modalities such as gene therapy, stem cell therapy, and retinal implants. 🌐 In this latest article, we explore: 🔑 Key Drivers: Rising demand for innovative therapies, including CRISPR technology and optogenetics  🌍 Regional Insights: The growth trajectory in North America, Europe, and APAC  💡 Market Opportunities: The potential for gene therapy and personalized treatments to revolutionize care for patients with retinitis pigmentosa While challenges like high treatment costs remain, the future looks promising for vision restoration and ophthalmic innovations. 🔗 Read the full article here to learn about the trends, drivers, and competitive landscape shaping the Retinitis Pigmentosa Treatment Market:  👉 Retinitis Pigmentosa Treatment Market Growth and Forecast 2024-2028: https://lnkd.in/gxzsDmzk #RetinitisPigmentosa #GeneTherapy #StemCellTherapy #RetinalImplants #OphthalmicTreatments #MedicalInnovation #VisionRestoration #HealthcareMarket #Technavio #MarketGrowth

Novartis has acquired Kate Therapeutics, a San Diego-based #genetherapy company, in a deal valued at up to $1.1 billion. Kate Therapeutics specializes in #preclinical programs targeting Duchenne muscular dystrophy (#DMD), facioscapulohumeral dystrophy (#FSHD), and myotonic dystrophy type 1 (#DM1). The company employs its proprietary DELIVER platform, which utilizes directed evolution of adeno-associated virus (#AAV) capsids to create #liver-#detargeted, muscle-tropic #vectors. Acknowledging recent challenges in muscular dystrophy treatments, such as Pfizer's investigational gene therapy for DMD failing to demonstrate efficacy in a Phase 3 trial, Novartis CEO Vas Narasimhan emphasized the company's commitment to learning from past experiences in the #musculardystrophy domain. By acquiring Kate Therapeutics, Novartis aims to leverage innovative #delivery #technologies to overcome previous obstacles in gene therapy for #neuromuscular #diseases. Muscular Dystrophy Association Muscular Dystrophy UK Muscular Dystrophy Canada Facioscapulohumeral Muscular Dystrophy Duchenne UK Duchenne Heroes #genetherapy #cellandgenetherapy #biotech #pharma #geneticmedice #biopioneers #innovation #geneediting https://lnkd.in/giRnS_6G