RNA Leaders

🧬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 Wikström 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

🧬RNA Leaders Industry Update🧬 ⭐Fantastic to see that Wave Life Sciences have announced positive interim data from ongoing Phase 2 FORWARD-53 study of an exon skipping oligonucleotide, WVE-N531, in boys with Duchenne muscular dystrophy. 🔗 https://lnkd.in/erzxGtSH ⭐ Congratulations to the CAMP4 Therapeutics team as they announce their plans for an IPO. 🔗 https://lnkd.in/edT8tNcs ⭐ Aptadir Therapeutics has launched, focussing on a new class of RNA inhibitors – DNMTs Interacting RNAs (DiRs) - targeting cancer and genetic conditions with limited treatment options. Congratulations to the Aptadir team! 🔗 https://lnkd.in/eq4Hmsfq ⭐ Researchers at The Johns Hopkins University demonstrate that an optimised CRISPR-Cas13d system can be used to target the hexanucleotide GGGGCC repeat expansion in C9orf72, showing treatment potential for ALS and FTD. 🔗 https://lnkd.in/efi2VF7N #RNALeaders #CRISPR #oligonucleotide #regRNA

🧬RNA Leaders Industry Update🧬 ⭐ Congratulations to NanoVation Therapeutics™ who have entered a $600M partnership with Novo Nordisk this week. The collaboration will progress development of genetic medicines targeting cardiometabolic and rare diseases, leveraging NanoVation’s lcLNP delivery technology and Novo Nordisk’s research experience in targeted disease areas. 🔗https://lnkd.in/egd6W6Cu 🔬 Exciting to see researchers at Zhejiang University and Nanchang University achieve cytosolic delivery of siRNA in cancer cells, using cholesterol-rich exosomes for membrane fusion. 🔗 https://lnkd.in/edMiuy4u ⭐ Fantastic to see that Korro Bio, Inc. has partnered with Novo Nordisk to advance their OPERA platform enabling oligonucleotide-directed RNA editing for two undisclosed therapeutic targets – congratulations to the Korro Bio team! 🔗 https://lnkd.in/enDtPfXA 🔬 Researchers at Zhejiang University underline the ability of Cas13j to achieve efficient in vivo C-to-U and A-to-G conversions, and its future potential to treat disease. 🔗 https://lnkd.in/enZr8RcT ⭐ Congratulations to the team at Aurora Biosynthetics on its official launch, providing end-to-end GPM production of mRNA, pDNA and LNPs. 🔗 https://lnkd.in/e4fhMzMh ⭐ Eurofins Genomics US has announced the opening of a new GMP oligo manufacturing facility, with increased manufacturing capacity and efficiency. 🔗 https://lnkd.in/eh-vnRCF ⭐ Congratulations to Roquefort Therapeutics plc who have announced that its STAT-6 siRNA has shown efficacy in a validated experimental model of immunological disease. 🔗 https://lnkd.in/eqNfJz8f #RNALeaders #oligonucleotide #LNP #RareDisease #RNAediting

🧬RNA Leaders Industry Update🧬 ⭐ Exciting to see that researchers at the University of North Carolina at Chapel Hill School of Medicine uncover the role of miR-29 as a significant driver of aging. 🔗 https://lnkd.in/eQhPsTVs ⭐ Following their announcement of their exciting new collaboration with Eli Lilly and Company, HAYA Therapeutics appoints Jordan Shin as CMO – congratulations! 🔗 https://lnkd.in/e_WwYnYV ⭐ Congratulations to GSK who have announced positive headline data from a phase 2 trial of its seasonal influenza mRNA vaccine program. 🔗 https://lnkd.in/e66x9VUZ ⭐ Moderna share financial and program updates, with five respiratory vaccines achieving positive phase 3 results, and three expected to be submitted for approval this year. 🔗https://lnkd.in/escPMJpy ⭐ Fantastic to see AcuraStem have secured $4M grant funding from the California Institute for Regenerative Medicine (CIRM) to progress its UNC13A program against ALS, FTD and other neurodegenerative diseases. 🔗 https://lnkd.in/ey54Hm-X #RNALeaders #mRNA #oligonucleotide #ncRNA #microRNA

We announced today program and financial updates demonstrating progress and strategic prioritization of our mRNA pipeline at our annual R&D Day event. The updates include data readouts in our respiratory vaccine portfolio and a revised financial framework. Read more: https://lnkd.in/escPMJpy