RNA Leaders

??RNA Leaders Industry Update?? ? ?Happy Thanksgiving week!? Here is our roundup of RNA news this week.. ? ?? Researchers at?Shenzhen Institutes of Advanced Technology (SIAT) of the Chinese Academy of Sciences have developed a light-guided siRNA delivery system based on cyanobateria, CTPA/siCSF1R, to enable precise spatiotemporal delivery of siRNA. ??https://lnkd.in/e79DtA_j ? ? Exciting to see that Intellia Therapeutics, Inc. have announced US FDA RMAT designation of its CRISPR-based therapy, Nex-z, a single dose treatment for ATTR amyloidosis. ?? https://lnkd.in/eFZUhMn2 ? ??Massachusetts Eye and Ear in collaboration with CureVac have demonstrated suppression of vitreoretinal disease in rabbits treated with mRNA-encoded RUNX1-Trap. ??https://lnkd.in/eqpSJBVk ? ?Sarepta Therapeutics and Arrowhead Pharmaceuticals have announced a $500M+ collaboration and licensing agreement for four clinical-stage and three pre-clinical stage siRNA programs in muscle, CNS and rare pulmonary disorders. ?? https://lnkd.in/eGPtBwPD ? Hear more exciting updates from key leaders in RNA this spring at RNA Leaders Europe!.. ? ?? @Thorsten Stafforst, Hospital of the university of Tübingen - Sharing insights on the RNA editing space, its opportunities and challenges. ? ?? Michiel Lodder, 20Med Therapeutics - Exploring PNP-delivered mRNA vaccines against infectious diseases ? ?? Julien Barteau, UCL - Leveraging an mRNA therapeutic to correct glutathione metabolism to treat Argininosuccinic aciduria ? ?? Michelle Werner, Alltrna - Diving into tRNA biology, new disease areas and future clinical progression ? ?? View the full 2025 agenda here: https://lnkd.in/eA4EmcX8 ? #RNALeaders?#CRISPR #tRNA #mRNA #RNAdelivery

?? RNA Leaders Industry Updates?? ?? Researchers at Karolinska Institutet and Karolinska University Hospital have developed a new microscopy method, TRISCO, that enables detailed 3D RNA analysis at cellular resolution in whole, intact mouse brains. ??https://lnkd.in/eBzrC6jb ?? Francesca Storici and her research team and Georgia Institute of Technology have uncovered RNA's unknown role in DNA damage repair. They found that RNA can help cells repair double-strand break, a severe form of DNA damage. ??https://lnkd.in/d5WCr8kK ?? Exciting, Cleveland Clinic reports a new siRNA drug, Zerlasiran, has been shown to significantly lower Lp(a) levels by over 80% in a recent phase 2 clinical trial. This could lead to new treatment options for those at risk of heart disease. ??https://lnkd.in/eatbhVdX Join us in Basel this spring to uncover more exciting updates from our leading network of RNA experts... ??Paola Pozzi, Sofinnova Partners – sharing insights on RNA-based investments during an interactive investor forum. ??Said Francis, Moderna – discussing effective and successful partnerships among pharma and large biotechs across the RNA field. ??Julien Baruteau, UCL – exploring how to leverage an LNP-mRNA therapeutic to correct Glutathione metabolism. ??Marcel Blommers, Saverna Therapeutics AG – discussing how to utilise AI-based small molecule targeting of miRNA to resolve inflammatory diseases. ?? You can view the full speaker line-up for 2025 here:https://lnkd.in/eA4EmcX8 #RNA #siRNA #AIdrugdiscovery #LNPs

?? RNA Leaders Industry Updates?? ? Exciting to see that Arcturus Therapeutics has received FDA clearance to start its H5N1 pandemic flu vaccine clinical trial! ?? https://lnkd.in/eCGfNBUN. ?? Researchers at the University of Rochester in partnership with CANbridge Pharmaceuticals Inc. highlight new technique, Stitch RNA, which utilises ribozyme-activated mRNA trans-ligation to enable delivery of dysferlin and dystrophin genes to restore protein function and correct muscular dystrophy pathology in mice. ?? https://lnkd.in/e9JNkS2S ?? Shahroud University of Medical Sciences and Shahid Beheshti University of Medical Sciences researchers demonstrate expression patterns of four lncRNAs in medicated and unmedicated schizophrenia patients. ?? https://lnkd.in/ecrPrQN4 ?? Researchers at the New York Genome Center and New York University used CRISPR-Cas13 to edit RNA to understand the impact of knock down of around 6200 lncRNAs on cell fitness, across five human cell lines. The study discovered 46 universally essential and 778 context-specific essential lncRNAs. ?? https://lnkd.in/eyhqyDpY Join us in Basel this spring to uncover more exciting updates from our leading network of RNA experts... ?? Alan Whitmore, e-therapeutics PLC - Discussing the role of AI in oligo drug discovery & development ?? Arun Kumar PhD, SCPM, CEPI (Coalition for Epidemic Preparedness Innovations) - Sharing insights on next generation delivery approaches for mRNA ?? Kemi Olugemo, MD, FAAN, Korro Bio, Inc. - Exploring oligonucleotide promoted editing of RNA for Alpha-1 antitrypsin deficiency. ?? Long-Cheng Li, Ractigen Therapeutics - Delving into RNA activation - discussing how can leveraging RNAa restore expression of UTRN for treatment of DMD & BMD ?? View the 2025 agenda here! - https://lnkd.in/eG8-v_zi #RNALeaders #lncRNA #RNAdelivery #RNAediting #CRISPR

??RNA Leaders Industry Update?? ?? Exciting to see that HuidaGene Therapeutics has received FDA clearance to proceed with application of its CRISPR-Cas13 RNA editing therapy, HG202, to treat neovascular age-related macular degeneration. This will be the first CRISPR/Cas13 RNA targeting therapy to enter the clinic. ?? https://lnkd.in/e3jHbaQt ? Congratulations to Leal Therapeutics, who have announced a $45M funding round to progress their pipeline of ASO and small molecule therapeutics targeting CNS disorders. ?? https://lnkd.in/eyRTFU2i ? Fantastic to see that exteRNA has completed a $11M seed round to advance small molecule modulation of RNA processing to treat cancer. ??https://lnkd.in/ec8WrCuh Hear more exciting updates from key leaders in RNA this spring at RNA Leaders Europe!.. ?? Dominique Verhelle, NextRNA Therapeutics - Discussing a small molecule approach to target lncRNA to regulate disease causing lncRNA-RBP interactions ?? William Jia, Virogen Biotech - Sharing insights on self-amplifying mRNA vaccines for immune-oncology diseases ?? Long-Cheng Li, Ractigen Therapeutics - Delving into an RNAa approach to restore expression of UTRN for treatment of DMD & BMD ?? Erik Digman Wiklund, Circio Holding ASA - Exploring circRNA expression to boost gene therapy ?? View the full 2025 agenda here: https://lnkd.in/eA4EmcX8 #RNALeaders #CRISPR #RNAa #mRNA #lncRNA

??RNA Leaders Industry Update?? ?? Exciting to see that Moderna and Merck have initiated their Phase 3 trial INTerpath-009, which evaluates Moderna's investigational neoantigen therapy, mRNA-4157 in combination with Merck's anti-PD-1 therapy, permbrolizumab in patients with non-small cell lung cancer. ?? https://lnkd.in/e5cNpE8W ?? Researchers at the Centre for Eye Research Australia (CERA) have used CRISPR-Cas13 to supress VEGF in human retinal cells for the first time, highlighting new alternative to eye injections in the treatment of macular degeneration and diabetic eye disease. ??https://lnkd.in/e9VYWKtm ? Congratulations to the team at Epitopea who have closed a $31M pre-series A financing round, to develop off-the-shelf RNA immunotherapies. ??https://lnkd.in/etU9YDA8 Hear further updates from experts in the RNA field, and make valuable connections at RNA Leaders Europe 2025! ???Join Samir Ounzain, HAYA Therapeutics for deep dive into the latest developments in ncRNA, in a dedicated workshop session this spring. ??In the meantime, check out our interview with Samir at the recent RNA Leaders USA 2024 meeting in San Diego, where he shares his outlook on the advancements and challenges in the RNA field, off the back of HAYA's exciting partnership announcement with Eli Lily! #RNALeaders #mRNA #RNAcollaboration #ncRNA #CRISPR

??RNA Leaders Industry News?? ?? Exciting to see that the Type 1 Diabetes Grand Challenge has announced funding for a research project exploring the influence of micoRNAs on beta-cell function in type 1 diabetes, the study will be led by Imperial College London and Helmholtz Munich. ?? https://lnkd.in/eEpb3Cjp ?? Researchers at Stanford University demonstrate that gold-siRNA supraclusters can be used to enhance the antitumour immune response of stereotactive ablative radiotherapy in mice models of head and neck cancer. ?? https://lnkd.in/e2KM9EsW ?? Researchers at The University of Texas at Arlington and Stanford University have shown that analysis of cell-free RNA in urine can be used for early detection genitourinary diseases, including cancer. ?? https://lnkd.in/e2ZwygmN ??We are excited to launch the agenda for our next meeting- 4th RNA Leaders Europe 2025!?? Join us in Basel next spring to hear more exciting updates from key leaders in the RNA space.. ?? Marie Wikstr?m Lindholm, Silence Therapeutics plc - Discussing Phase 2 study of siRNA therapeutic, zerlasiran, in patients with elevated lipoprotein(a) ?? Dr. Myriam Mendila, CureVac - Exploring the clinical development of an mRNA based off-the-shelf cancer vaccine to treat Glioblastoma ?? Pad Chivukula, Arcturus Therapeutics, - Delving into the LUNAR platform and progression of clinical candidates for Cystic Fibrosis ?? James Hamilton MD, MBA, Arrowhead Pharmaceuticals - Sharing insights on Arrowheads clinical progression of RNAi candidates ARO-INHBE & ARO-ALK7 to tackle obesity ?? You can view the full speaker line-up for 2025 here: https://lnkd.in/eA4EmcX8 #RNALeaders #microRNA #mRNA #siRNA #siRNA

??RNA Leaders Industry Update?? ? Exciting to see that Wave Life Sciences have announced first-ever therapeutic RNA editing in humans. Their ongoing Phase1b/2a RestorAATion-2 study of WVE-006, a GalNAc conjugate A-to-I editing oligonucleotide, in patients with alpha-1 antitrypsin deficiency (AATD) produced positive proof of mechanism data. ?? https://lnkd.in/eVgYjhdx ?? Researchers at Hebei Medical University show that intratracheal nebulization of LNP-siRNAs targeting integrin α5β1, significantly decreased Staphylococcus aureus-induced pneumonia in the lungs of infected mice, highlighting a new therapeutic avenue for respiratory infections. ??https://lnkd.in/ewkPzMhw ? The therapeutic potential of CRSIPR-Cas9 ribonucleoprotein-LNP complexes has been expanded by researchers at University of California, Berkeley. Their study observed a large increase in genome-editing levels in the liver and lungs of mice upon single intravenous injections of iGeoCas9 RNP-LNP complexes, compared to previous viral or nonviral delivery methods. ??https://lnkd.in/eh6dpJPS #RNALeaders #RNAediting #GalNAc #LNP #RNAdelivery

??RNA Leaders Industry Update – microRNA Spotlight ?? ??We reflect on a very exciting week for microRNA!??Fantastic to see Victor Ambros and Gary Ruvkun awarded The Nobel Prize in Physiology or Medicine for their discovery of microRNA and its role in post-transcriptional gene regulation – congratulations! ???? ??https://lnkd.in/d6mnFtXH ??Judo Bio has launched with $100M Series A financing, to further develop its STRIKE platform which enables cell specific delivery of conjugated siRNA to the proximal tubule of the kidney. ?? https://lnkd.in/giEF8tnM ?? Eli Lilly and Company and insitro enter a collaboration to advance medicines for metabolic diseases, leveraging insitro's AI/ML-based platform for target identification. ?? https://lnkd.in/giv2g2V9 ?? RNAi biotech, City Therapeutics launches with $135M Series A funding, focussing on building an siRNA-engineering platform and designing RNAi triggers, to improve potency, efficacy and specificity of RNAi-based medicines. ?? https://lnkd.in/gsrG_Uw7 ?? Researchers at the Karolinska Institutet and Chinese Academy of Medical Sciences have discovered that lncRNA, SNHG26, plays a critical role in wound healing, where mice lacking SNHG26 showed delayed wound healing. Highlighting the potential of SNHG26 targeting as therapeutic tool for acute and chronic wounds. ??https://lnkd.in/gSVYyQmY #RNALeaders #microRNA #nobelprize

BREAKING NEWS The Nobel Assembly at the Karolinska Institutet has today decided to award the 2024 Nobel Prize in Physiology or Medicine to Victor Ambros and Gary Ruvkun for the discovery of microRNA and its role in post-transcriptional gene regulation. This year’s Nobel Prize honours two scientists for their discovery of a fundamental principle governing how gene activity is regulated. The information stored within our chromosomes can be likened to an instruction manual for all cells in our body. Every cell contains the same chromosomes, so every cell contains exactly the same set of genes and exactly the same set of instructions. Yet, different cell types, such as muscle and nerve cells, have very distinct characteristics. How do these differences arise? The answer lies in gene regulation, which allows each cell to select only the relevant instructions. This ensures that only the correct set of genes is active in each cell type. This year’s medicine laureates Victor Ambros and Gary Ruvkun were interested in how different cell types develop. They discovered microRNA, a new class of tiny RNA molecules that play a crucial role in gene regulation. Their groundbreaking discovery revealed a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans. It is now known that the human genome codes for over one thousand microRNAs. Their surprising discovery revealed an entirely new dimension to gene regulation. MicroRNAs are proving to be fundamentally important for how organisms develop and function.

??RNA Leaders Industry Update?? ?Congratulations to CAMP4 Therapeutics and BioMarin Pharmaceutical Inc. recent research collaboration to advance novel regRNA targeting medicines for rare genetic diseases. ?? https://lnkd.in/ekkqn6BQ ? Great to Shape Therapeutics Inc. launch their DeepREAD AI platform which has been created for precision RNA editing. AI model can be used to enable efficient design of ADAR guide RNAs for any target. ?? https://lnkd.in/edEN79zw ? Researchers at the?University of Pennsylvania have demonstrated with new mice data that they can successfully target protein related to C. difficile and protect the mice from the infection using mRNA technology in a multipronged vaccine design. ?? https://lnkd.in/eiXVmWQF ? ? Great to see that BioNTech SE and Google DeepMind have teamed up to build an AI lab assistant to aid researchers in planning scientific experiments and help better predict their outcomes. ??? https://lnkd.in/eS4TKDcp ? The team at the Institute for RNA Innovation, University of Pennsylvania has developed the first norovirus vaccine candidate utilizing mRNA-LNP platform technology. The bivalent vaccine has so far shown it can effectively protect human enteroids from infection by the most prevalent genotype (GII.4). ?? https://lnkd.in/eEaeh7yM #RNA #RNAediting #AI #regRNA #LNP #mRNA