Accurate clinical translation of preclinical research remains challenging, primarily due to species-specific differences and disease and patient heterogeneity. Given this, how do you know how relevant your model is? This study by Javelin Biotech describes the development of pathway-based similarity measurement (PBSM), which leverages RNA-seq data and pathway-based information to assess the human relevance of preclinical models for specific CoU. This figure shows PBSM-based scores for pre-clinical animal models of nonalcoholic fatty liver disease compared to reference human NAFL liver tissue samples for the KEGG NAFL pathway. There is a room for improvement - Stay tuned for more about Javelin’s human healthy and disease models! bioRxiv preprint: https://lnkd.in/dUZVAGCD
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📢 We are excited to present our new collection, "Exploring Novel In Vitro Models for Cystic Fibrosis Research," now open for submissions! 🚀🔬 https://lnkd.in/d75Z3Rkq Cystic fibrosis (CF) is a complex genetic disorder affecting the respiratory and digestive systems, often leading to chronic lung inflammation and pathogen infections. Current animal models face limitations in replicating CF's human complexities, highlighting the need for innovative in vitro approaches. With the recent approval of the “FDA Modernization ACT 2.0,” the use of complex in vitro cultures for drug testing is now a reality, offering more accurate research alternatives. This collection, led by Dr. Roberto Plebani, Dr. Yuncheng M. and, Dr. Domenico Mattoscio, welcomes articles focusing on novel in vitro models for CF research, with a particular interest in papers exploring 3D in vitro models, like organoids and organ-on-a-chip technology, which can enhance drug delivery predictions and provide insights into inflammation dynamics. 📃 Register your interest here: https://lnkd.in/di2q-s58 Manuscript Submission Deadline: 31 January 2025 #CysticFibrosis #CFResearch #InVitroModels #Organoids #OrganOnAChip #DrugDiscovery #Pharmacology #Research #InnovativeScience
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There are so many rodent models available for preclinical target and drug discovery for #MASH. But which model offers the highest resemblance of the human disease? The answer is simple. In the recent issue of Nature Metabolism, the renowned #LITMUS consortium concludes that Gubra’s GAN DIO-MASH mouse model ranks #1 for human proximity, a key indicator of clinical translatability, in terms of both metabolic relevance and ability to induce MASH-fibrosis. LITMUS compared a wide range of the most commonly used preclinical MASH models, evaluating their metabolic phenotype, liver histopathology and transcriptomics against corresponding MASH patient data. We are very proud and grateful to be recognized by LITMUS as the provider of the most translational preclinical model. Check out the paper and explore the objective ranking of preclinical models of MASH in the industry. #PreclinicalModel #PreclincalResearch #ClinicalTranslatability #MASLD #Publication #PreclinicalCRO #ResearchModels
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Sometimes, proteins your body makes can cause or contribute to disease. These proteins can be either be faulty, or healthy. RNAi therapeutics are a type of medicine, pioneered by Alnylam, that treats disease by targeting and disabling specific mRNA (messenger RNA) which tell your body how to make these unwanted proteins. #ScienceCurious to learn more about the science of #RNAi? Click here: https://bit.ly/3oR8r2l #RNAiTherapeutics #siRNA #GeneSilencing #science
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The new Fragment Sequencing approach developed by researchers at ETH Zurich is opening up new insights into single-cell transcriptomics, and expanding the scope of what we know about cell-to-cell interaction, disease progression and tumorigenesis. Read our summary of the study to learn more : 👉 https://lnkd.in/dRQDGZ6N #BDBiosciences #SingleCellAnalysis #SingleCell #SpatialBiology #Rhapsody #BD
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The new Fragment Sequencing approach developed by researchers at ETH Zurich is opening up new insights into single-cell transcriptomics, and expanding the scope of what we know about cell-to-cell interaction, disease progression and tumorigenesis. Read our summary of the study to learn more : 👉 https://lnkd.in/dRQDGZ6N #BDBiosciences #SingleCellAnalysis #SingleCell #SpatialBiology #Rhapsody #BD
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🙌 I am thrilled to share that Ochre Bio, one of our innovative portfolio companies, has entered into a groundbreaking, up to $1 billion partnership with Boehringer Ingelheim to advance treatments for metabolic-associated steatohepatitis (MASH), a severe form of liver disease. 🧬 Ochre Bio is at the forefront of chronic liver disease medicine development and RNA biotechnology, developing therapies that harness the liver's regenerative capabilities to combat chronic liver diseases. This collaboration will leverage Ochre Bio's expertise, proprietary discovery platform in liver disease research, genomics, and machine learning to target disease pathways effectively, offering new hope in a challenging therapeutic area. 👏 As an investor, it's incredibly rewarding to support and be part of solutions that promise significant impacts on patient health globally. This partnership not only underscores Ochre Bio's potential but also marks a significant step towards transformative healthcare solutions. 🥂Congratulations to the team at Ochre Bio! I can't wait to see more innovation and see how we can make a meaningful difference in the lives of those affected by chronic liver disease. #Biotech #HealthcareInnovation #Partnership #VentureCapital #Biotechnology #VC #LiverHealth #RNAtherapy #BiotechInvestment #HealthTech #InnovationInHealthcare #ChronicDiseaseManagement #MedicalResearch #BiotechVC #StartupGrowth #CollaborativeInnovation #FutureOfMedicine
🎉 Announcing our new partnership with Boehringer Ingelheim to discover and develop novel regenerative treatments for chronic liver disease! Our multi-year collaboration will focus on using our proprietary discovery platform – which combines advanced genomics, deep phenotyping and machine learning – and human-organ translational models to accelerate the development of novel targets for undertreated liver diseases, such as late-stage metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis. The deal brings in up to $35m in upfront and near-term milestone payments, with late preclinical, clinical, regulatory and commercial milestones with the potential to exceed $1B, as well as tiered commercial royalties. Chronic liver disease (CLD) and associated cirrhosis accounts for approximately 1 million deaths per year. Currently there are no approved medicines for cirrhosis resulting from CLD and there is an urgent need for new effective treatment options. The partnership is part of Boehringer Ingelheim’s commitment to improving the lives of people living with liver diseases. Together we will continue to pioneer liver regeneration research, with a shared vision of reversing the progression of diseases that affect millions worldwide. Read our full announcement ➡️ https://lnkd.in/ePFjWumn #TeamOchre #biotech #genomics #LiverDisease
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We've built the first truly multimodal real-world clinical dataset purpose-built to capture the underlying biology of cardiometabolic diseases, including the role of GLP-1s in treatment. This dataset is designed to help drug developers and researchers: ▶ Understand the patient journey for those on GLP-1s ▶ Quantify any side effects associated with GLP-1 use ▶ Demonstrate therapeutic effects beyond intended use including secondary benefits derived from additional modalities and exploratory use ▶ Compare efficacy of products that were never studied head-to-head across a range of different real-world measures ▶ Develop precision medicine tools that identify patients with uncontrolled symptoms and/or match patients to the right treatments Our data go deep: drawing in imaging, waveforms, notes, pathology, and videos linked to long-term outcomes from the EHR for GLP-1 users and matched control groups. We'll be at the GLP-1 Based Therapeutics Summit on May 15-16, 2024 in Philadelphia, PA to meet and learn from others who are working in this space. We hope to see you there! Schedule a time to chat: https://lnkd.in/g23jwAf5 See the full agenda: https://ter.li/86n56l Visit our GLP-1 page: https://lnkd.in/g4AuzutP
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A concise overview of lipid nanoparticles for RNA delivery: Yutian Ma at University of North Carolina at Chapel Hill, Yupeng Chen at National Center for Nanoscience and Technology and co-authors reviewed LNP formulation and preparation methods, followed by comprehensive characterization, including size, polydispersity index (PDI), charge, surface morphology, RNA encapsulation efficiency and stability. Their article also delved into in vitro cell experiments, covering cell selection, viability, cellular association/uptake, endosomal escape, and efficacy https://lnkd.in/eP9UM9za Lastly, they explored in vivo animal experiments to assess LNP performance, including animal selection, administration, dosing, safety, and therapeutic efficacy. An article also authored by Shiyao Li and Xin Lin #LNP #drugdelivery #vaccines #RNA #nanoparticles #Vesiculab
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"In a major step forward in drug development, researchers at the National Institute of Standards and Technology (NIST) have developed a tool for building a system known as heart-on-a-chip (HoC). This technology seeks to address the limitations of conventional cardiovascular drug development, which relies heavily on animal testing. By replicating humanlike models for studying cardiovascular diseases, the HoC system holds the promise of helping to replace animal testing, shortening drug development timelines, and reducing costs. The NIST team's results were published in Lab on a Chip." #heartresearch
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Attended the Organoid Conference in Cambridge, where the research highlighted the significant potential of organoids in preclinical studies. Organoids are powerful tools for modeling human diseases and testing treatments accurately. They play a crucial role in drug screening, disease modeling, and understanding disease mechanisms. Such conferences are invaluable for staying at the forefront of research and are crucial for my career development in the field. #OrganoidResearch #PreclinicalStudies
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