Avidity Biosciences, Inc.’s Post

Did you know over 30% of clinical trials fail due to drug-induced organ damage? At TAmiRNA, we're part of the IMI2 TransBioLine project, working on the cutting edge of this challenge. We're exploring microRNA biomarkers as a way to detect drug-induced organ damage early, improving patient safety and drug development success rates. Our Senior Scientist, Kseniya Khamina-Kotisch, recently discussed our work in a "Meet the Expert" interview. She dives into TAmiRNA's key contributions in microRNA biomarker analysis and the development of the miND® pipeline for precise small RNA quantitation. Learn more about how our work is advancing patient safety and biomarker discovery: https://lnkd.in/ejnwJ5xg #drugdiscovery #drugtoxicity #mirna #biomarker #patientcare #TransBioLine

Over 90% of the population carries at least one genetic variant that could affect their response to medications.💊 Preclinical models often fail to capture human genetic variability, making it difficult to replicate complex drug responses that translate to clinical outcomes. Altis offers a biobank of human donors that enables more representative preclinical testing, improving predictions of drug responses across varied populations.👥 Read our latest publication to learn how Altis characterized its human intestinal stem cell biobank: https://hubs.ly/Q02L4Yt60

4D Molecular Therapeutics announced positive interim data from the phase 2 PRISM clinical trial evaluating intravitreal 4D-150 in wet age-related macular degeneration (#AMD) patients with severe disease activity and a high treatment burden during the #Angiogenesis, Exudation, and Degeneration 2024 #conference. The data was presented by Dr. Arshad M. Khanani, and it consisted of 24-week results from the randomized phase 2 dose expansion cohort of the #PRISM clinical trial. #AMD #wAMD #MacularDegeneration #IntravitrealInjection #IVI #IVT #neovascularAMD #nAMD 

The Nanoparticles for RNA Therapeutics Special Feature focuses on RNA therapeutics and their delivery, highlighting the diverse approaches used to discover and characterize delivery systems. The articles focus on the delivery of RNA to new tissue types, the development of self-reporting RNA nanosystems, a description of novel delivery systems, and fundamental biological studies on how delivery systems can be engineered with improved efficacy. Discover more in the PNAS Special Feature: https://ow.ly/nprR50R0Ep6

AUG 1st marks World RNA Day (pun intended) In recent years, RNA has gained prominent attention as a therapeutic molecule, playing an important role in drug discovery. Several RNA-based medications have been already approved for clinical use, while others are still under investigation or preclinical trials. These range from RNA interference (RNAi), antisense oligonucleotides (ASOs), and circular RNAs to messenger RNAs (mRNAs). In this way, RNA can work to selectively function on previously-thought undruggable proteins, transcripts, and genes, broadening the range of therapeutic targets. This paves the way for the development of novel treatments against many rare genetic diseases but also oncologic diseases. At InnoSer, we have worked with RNA-based therapeutics in multiple disease models. Find out how our expertise in working with RNA-based therapeutics can help accelerate your research now: https://lnkd.in/eHH4bmZT #RNADay #AUG #RNATherapies #RNAi #mRNA #ASO

🤗 Find us at Booth 517, Festival of Biologics 2024 in San Diego from April 15-17. Don't miss Xiaoyuan Ran's talk on "Transforming Tumor Research: Engineering MHCs as Versatile Reagents" today at 3pm. 📊 Poster Presentations: Explore our innovative research through five engaging posters: #77: Transforming Tumor Research: Engineering MHCs as Versatile Reagents #78: GPCR VLPs & Nanodiscs for Drug Discovery #79: Tailored AAV Solutions: ELISA Kits and Customized Variant Kit Development #80: Enhancing Cell Therapy with GMP-Grade Cas9 Nuclease Exhibiting Superior Quality and Batch Consistency in Gene Editing #81: The Second-Generation High-Fidelity Cytosine Base Editor AccuBaseTM Can Proficiently Modify Multiple Genes Within A Human Primary T cell While Minimizing Off-target Effects #KACTUS #festivalofbiologicsUSA #Biotech #Innovation #DrugDiscovery #antibodytherapeutics #antibodydiscovery

Unlocking the Potential of RNA Editing: A Promising New Frontier in Genetic Medicine: In a remarkable scientific breakthrough, RNA-editing drugs are rapidly advancing from the preclinical stage into early-stage clinical trials, offering new hope for patients with genetic disorders. This emerging field of genetic medicine holds immense promise, as these innovative therapies harness the power of RNA editing to directly address the underlying genetic defects, rather than merely managing symptoms. The article in Nature highlights several promising RNA-editing drug candidates that have entered clinical trials, targeting a diverse range of genetic conditions, including Duchenne muscular dystrophy, cystic fibrosis, and other rare disorders. The successful development and approval of these therapies could pave the way for a new era in personalized genetic medicine, where targeted treatments become the norm, transforming the lives of countless individuals affected by genetic disorders. As these RNA-editing drugs progress through clinical trials, the scientific community and the broader public eagerly await the outcomes. The potential of these groundbreaking therapies to address the root cause of genetic conditions and provide new treatment options for patients with limited or no effective therapies available is truly exciting. The future of genetic medicine has never been brighter. #Cancer #rna #genetics #dna #oncology #vaccine #clinicaltrials #drugdiscovery #innovation #genetherapy #medicine #genomics

Highlight Content of the Month (November 2023) . If you miss out some insights from us, check it out on this post!  Discover insights and stay updated.  . 1. In vivo vs in vitro drug testing : What are the differences that you need to know?  Link : https://lnkd.in/dWh2_PbA  2. MSC therapy in oncology current status and future development of MSC therapy Link : https://lnkd.in/dpqBMub4  3. Where can doctors obtain MSCs from our body? Link : https://lnkd.in/dA4rH5cv  4. Unlocking the secret tips to efficient MSC cell culture Link : https://lnkd.in/dbdmS6_u  5. Exploring the Future of Anti-Cancer Innovations with MSC-Based Gene Editing Link : https://lnkd.in/dyXgrDJQ  . Tag your connections to learn, gaining insights and exchange some useful information that can empower both your knowledge and career. . Looking to find deeper insights on MSCs, cell therapy, and more check out our blog:  https://lnkd.in/gcPXfAVq #Atlantis #BenchtoBed #AtlantisBioscience #DiscoverSolutions #TranslationalSolutions #AtlantisBioscience #DiscoverSolutions #TranslationalSolutions #CancerInnovations #GeneEditing #MSCs #CRISPRCas9 #AntiCancerSolutions #CuttingEdgeResearch #BiotechAdvancements #ScientificBreakthroughs #MSCBasedGeneEditing #GeneEditing #MSCs #AntiCancer #CancerResearch #Biotechnology #InnovativeTherapies #ResearchBreakthroughs #StemCells #PrecisionMedicine #CancerTreatment #GeneticEngineering #BiotechAdvancements #ScientificAdvancements #HealthInnovation #Immunotherapy #CellandGeneTherapy #FutureofMedicine #SeneScenceMatters

August 01st is World RNA Day! 🧬 But do you know why it's celebrated on this date? 🤔 Every strand of RNA is a sequence of four building blocks called nucleotides. The first three are adenine, uracil, guanine - A, U, and G. Put together in this order, these three nucleotides form a ‘codon’ that instructs the ribosome to start building the protein. Since this represents the starting instruction, so AUGust 01st has become associated with celebrating RNA!¹ Ready to learn more about RNA and it's importance for oncology research? Read our informative blog posts to explore RNA analysis methods, transcriptomic technology for mRNA profiling studies, and RNA-Seq Panels for preclinical drug development. 📄Choosing the Right RNA Analysis Methods: Genome-wide RNA-Seq vs. NanoString nCounter® vs. Targeted RNA-Seq Panel ➡️https://hubs.la/Q02JMf9m0 📄Genome-Wide RNA-seq and Array-Based NanoString Transcriptomic Technologies: Which to Use and When? ➡️ https://hubs.la/Q02JMf9l0 📄Leveraging Targeted Mouse I/O RNA-Seq Panels to Improve the Translatability of Immuno-Oncology Studies ➡️https://hubs.la/Q02JM2dm0 #WorldRNADay #RNAday #RNA #RNASeq ¹https://hubs.la/Q02JM6hx0

Join us today to learn how to mine oncology multi-omics data to quickly identify candidate targets and biomarkers relevant to immuno-oncology. https://lnkd.in/eGSTvzU6