Today at #ASGCT we will be sharing additional data from our team at Vesigen Therapeutics, Inc., including #targeted #delivery of #genomeeditors to a variety of cells, which we hope will enable new therapeutic strategies for diseases with unmet need. Please come by our posters, listed below, if interested in learning more: ARMMs as Non-Viral Vehicles for the Delivery of Therapeutic Payloads to Liver Sinusoidal Endothelial Cells Abstract Number: 1238 Fractionation of distinct extracellular vesicle populations by charge and density to evaluate the heterogeneity and purity of ARMMs Abstract Number: 1246 ARMMs as a Versatile and Modular Non-Viral Platform for the Functional Delivery of Genome Editors Abstract Number: 1247 Engineering ARMMs with Modular T Cell-Specific Engagers for Non-Viral Delivery of Therapeutic Payloads Abstract Number: 1248 Engineering Directed Tropism in the Non-Viral ARMMs Delivery Platform Abstract Number: 1249 #nonviral #invivo #drugdelivery #genomeediting
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𝗜𝘀 𝗥𝗡𝗔 𝘀𝘁𝗿𝘂𝗰𝘁𝘂𝗿𝗲 𝘁𝗵𝗲 𝗵𝗶𝗱𝗱𝗲𝗻 𝗸𝗲𝘆 𝘁𝗼 𝗯𝗿𝗲𝗮𝗸𝘁𝗵𝗿𝗼𝘂𝗴𝗵 𝘁𝗵𝗲𝗿𝗮𝗽𝗶𝗲𝘀? Behind every RNA-based treatment, a complex folding process can either drive success or lead to failure. In our latest eBlog, we reveal how RNA’s intricate structure influences the stability of vaccines, the success of small molecule drugs, and the power of siRNA therapies. The answers could redefine how we design the next generation of RNA medicines. Discover the science behind it all in our eBlog! https://lnkd.in/gJpw8Yza #RNAInnovation #Therapeutics #Genomics #Biotech #RNAstructure #Eclipsebio
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For more than a decade, single-cell RNA sequencing, or scRNA-seq, has played a critical role in identifying the molecular mechanisms driving complex diseases and, in turn, pinpointing the specific types of cells for which a drug works. But analyzing scRNA-seq data has never been straightforward, limiting its practical utility to researchers. To harness the full potential of human cell data to create better therapies, we need to translate scRNA-seq to the functional level by annotating de novo cell types and building cell-type references researchers can actually use. My ZS colleagues Thomas Skot Jensen, Pascal Timshel, Christina Bligaard Pedersen and Francisco Avila Cobos describe how creating a periodic table of cells can help unlock the therapeutic potential of single-cell-based approaches. https://lnkd.in/di_iUbu8 #pharma #drugdiscovery
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Oligos roll on with Novo Nordisk teaming up with Korro Bio... We expected mRNA to to slow down due to delivery challenges (see our 2024 advanced therapies predictions) and lead to some tailwinds for oligos, but this is another level. Novo Nordisk has entered a $530 million biobucks deal with Korro to collaborate on two cardiometabolic-related RNA targets. Korro will advance the programs through preclinical development, with Novo taking over for clinical studies. The partnership leverages Korro's OPERA platform for RNA editing, and Novo will focus on exploring new cardiometabolic treatment approaches. Oligos are increasingly going after broader indications and are no longer limited to rare disease. Novel chemistries and delivery capabilities are helping move this along. Want to know more? We'll cover some of this on our webinar on Thursday. Reach out to register. #biotech #RNA #drugdiscovery
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All RNAs fold into complex structures that regulate RNA biology and the effectiveness of RNA-targeting and RNA-based medicines. In this month's eBlog I review some of the key aspects of RNA structure and how it can affect the efficacy of RNA vaccines, small molecule drugs, and small oligonucleotide therapies.
𝗜𝘀 𝗥𝗡𝗔 𝘀𝘁𝗿𝘂𝗰𝘁𝘂𝗿𝗲 𝘁𝗵𝗲 𝗵𝗶𝗱𝗱𝗲𝗻 𝗸𝗲𝘆 𝘁𝗼 𝗯𝗿𝗲𝗮𝗸𝘁𝗵𝗿𝗼𝘂𝗴𝗵 𝘁𝗵𝗲𝗿𝗮𝗽𝗶𝗲𝘀? Behind every RNA-based treatment, a complex folding process can either drive success or lead to failure. In our latest eBlog, we reveal how RNA’s intricate structure influences the stability of vaccines, the success of small molecule drugs, and the power of siRNA therapies. The answers could redefine how we design the next generation of RNA medicines. Discover the science behind it all in our eBlog! https://lnkd.in/gJpw8Yza #RNAInnovation #Therapeutics #Genomics #Biotech #RNAstructure #Eclipsebio
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🌐𝐄𝐱𝐩𝐥𝐨𝐫𝐢𝐧𝐠 𝐁𝐫𝐞𝐚𝐤𝐭𝐡𝐫𝐨𝐮𝐠𝐡𝐬 𝐢𝐧 𝐑𝐍𝐀 𝐃𝐞𝐥𝐢𝐯𝐞𝐫𝐲 & 𝐎𝐥𝐢𝐠𝐨𝐧𝐮𝐜𝐥𝐞𝐨𝐭𝐢𝐝𝐞𝐬🌐 As the field of RNA therapeutics continues to evolve, we're witnessing transformative developments, particularly in RNA delivery systems. 📈 🔹 Non-Viral Systems dominate the landscape, driven by advances in technology and increasing regulatory support. Their growth surged during the COVID-19 pandemic and has since stabilized. At the same time, Non-viral Systems exhibit high entry and exit rates as researchers work to overcome challenges such as efficiency and specificity, leading to a dynamic pipeline 🔹 In contrast, Viral Systems are gaining much more cautious growth, The recent rise in interest and funding since 2022 shows a shift towards embracing the high-reward potential of these systems, despite safety and scalability concerns. Looking ahead, it’s clear that RNA delivery systems are set for further innovation, promising impactful therapies for unmet medical needs. Follow us at Hanson Wade Intelligence to stay tuned for more analysis like this! Our analysis was made possible with drug and clinical trial data from Beacon #RNATherapeutics #Oligonucleotides #Biotechnology #DrugDevelopment #PharmaInnovation #NonViralDelivery #ViralDelivery #BiotechTrends #TherapeuticDelivery #RNA
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We are thrilled to share that Leveragen has entered into a multi-target research, option and license agreement with Moderna to advance therapeutic solutions. This collaboration will utilize our unique Singularity Sapiens Mouse, a next-generation genetic model engineered to exclusively produce single-domain antibodies (sdAbs) from the complete human VH repertoire. These fully human sdAbs offer unparalleled diversity, affinity, and developability, making them ideal for developing a wide range of biological modalities. By integrating Leveragen's cutting-edge sdAb technology with Moderna’s pioneering mRNA platform, this partnership aims to create novel treatments targeting diseases with unmet medical needs. We are eager to provide further updates as we move forward in this transformative collaboration. https://lnkd.in/gzykEHRA #Biotech #Leveragen #Moderna #mRNA #SingleDomainAntibodies #Nanobodies #Hcab #VHH #AntibodyTherapeutics
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𝐀𝐥𝐥𝐨𝐠𝐞𝐧𝐞 & 𝐀𝐫𝐛𝐨𝐫 𝐏𝐚𝐫𝐭𝐧𝐞𝐫 𝐟𝐨𝐫 𝐍𝐞𝐱𝐭-𝐆𝐞𝐧 𝐀𝐥𝐥𝐨𝐂𝐀𝐑 𝐓 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐢𝐧 𝐀𝐮𝐭𝐨𝐢𝐦𝐦𝐮𝐧𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 Allogene Therapeutics, Inc. (Nasdaq: ALLO) Allogene Therapeutics and Arbor Biotechnologies, Inc. Arbor Biotechnologies announce a global gene editing licensing agreement for Allogene's next generation AlloCAR T platform for autoimmune disease (AID) treatment. Zachary Roberts, M.D., Ph.D. Zachary Roberts, of Allogene, highlights the potential of CAR T therapy for AID and the importance of differentiation for success. The agreement grants Allogene access to Arbor's CRISPR gene-editing technology for developing effective CAR T approaches. Allogene's expertise in CAR T R&D aims to create next-generation allogeneic CAR T products with reduced or chemotherapy-free conditioning. Their first AID AlloCAR T investigational product is set to enter Phase 1 clinical trials in early 2025. Devyn Smith, Ph.D. Devyn Smith, CEO of Arbor Arbor Biotechnologies, emphasizes the collaboration's goal of leveraging gene-editing technologies to advance novel CAR T therapeutics for autoimmune diseases. #AllogeneTherapeutics #ArborBiotechnologies #geneediting #celltherapy #autoimmunedisease #CRISPR #clinicaltrials #biotechnology
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🤝 We, at Scailyte, are thrilled to announce our research collaboration with Visterra Inc., focused on utilizing AI-driven biomarker discovery to enhance the development of novel therapeutics for autoimmune diseases. 🔍 For more information: https://lnkd.in/dr2xHd4W #AutoimmuneDiseases #SingleCellTechnologies #Research #Innovation #Healthcare
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Biomarkers have gone beyond their original use as diagnostic tools and now touch every aspect of healthcare. Discovering new ones requires more insight than conventional single-cell analysis can provide, which is exactly why Mission Bio keeps adding new capabilities to its multiomics Tapestri platform. To see how Tapestri is playing an important role in biomarker discovery, check out this deep dive by Ashleen Knutsen for Genetic Engineering & Biotechnology News. https://lnkd.in/g8HVzyUt #biomarkers #multiomics
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Ensuring successful #oligonucleotidetherapeutics development demands specialized bioanalytical and CMC support. Experts are rare in this field. Finding labs with expertise in oligonucleotide analysis is a challenge and smaller #CROs often lack the necessary equipment. Don’t give up control. Choose Sannova: #boutiqueCRO services for personalized, transparent preclinical support. Expert Advice for #ASOs, siRNA and miRNA analysis, pharmacokintetic (PK), toxicokinetic (TK) studies. Quick turnarounds for fast progress and regulatory review. For a free demonstration of our capabilities in action download our brand new whitepaper, offering exclusive insights into siRNA quantification. #bioanalyticalexcellence
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