RNA Leaders

🧬RNA Leaders Industry Updates – End of Year Wrap🧬   Festive greetings to our RNA Leaders community. As the end of a brilliant year for RNA innovation approaches, take a look at some of the top clinical developments, new collaborations, fundraising successes and community gatherings in the field over the year. ⭐Download the RNA Leaders End of Year Newsletter  🔗https://lnkd.in/ejJPfueU And wrapping up the year with this week’s highlights! See you in 2025! ⭐Congratulations to Ionis Pharmaceuticals, Inc. on the approval of TRYNGOLZA™ (olezarsen) in the USA. It is an antisense oligonucleotide and it will be the first-ever treatment approved for adults living with familial chylomicronemia syndrome as an adjunct to diet. 🔗https://lnkd.in/eg2G4NpH ⭐ Researchers at University of South Carolina have completed a recent study into how emotional experiences shape behavior highlighting the critical role of long noncoding RNAs. 🔗https://lnkd.in/eCXCh88i ⭐Congratulations to Evonik and Silexion Therapeutics Corp for entering a collaboration on siRNA formulation development and progressing Silexion’s efforts on targeting KRAS. 🔗https://lnkd.in/edeuQnWu ⭐AGS Therapeutics expands its leadership team by appointing Dr. Kristin Luther to drive CMC and analytics. 🔗https://lnkd.in/eBSFYFpc #lncRNA #KRAS #siRNA #Antisense #CMC #RNAinnovation

🧬RNA Leaders Industry Update🧬   ⭐Researchers at University of Pennsylvania have investigated a new approach to treat pre-eclampsia, via LNP-based delivery of VEGF mRNA to the placenta of mice. Treated mice saw improved maternal blood pressure and fetal outcomes. 🔗https://lnkd.in/eyE-yPjv   ⭐ Fantastic to see that Sirius Therapeutics has announced promising data from its Phase 1 first-in-human trial of SRSD107, a siRNA therapeutic for treatment of thromboembolic disorders. 🔗https://lnkd.in/euDvAKJK   ⭐Congratulations to ProQR Therapeutics, who have announced new funding of $8.1M from Rett Syndrome Research Trust to advance their RNA editing therapeutic, AX-2402, into clinical trials. 🔗https://lnkd.in/ePg7Z8jA   ⭐Charles Kunsch has been appointed to NextRNA Therapeutics' board of directors this week - congratulations! 🔗https://lnkd.in/ez4HxV6Y Hear more exciting updates from RNA experts this March 4-6th at RNA Leaders Europe 2025!... 🎤 Harris Makatsoris, King's College London - Sharing insights on sustainable, scalable manufacturing of mRNA therapeutics and vaccines. 🎤 Trevor Hallam, Sail Biomedicines - Exploring Endless RNA, preclinical data and movement into the clinic. 🎤 Hari Pujar, Tessera Therapeutics - Delving into next generation RNA gene writing to treat cancer and monogenic diseases. 🎤 Steve Pascolo, Universitätsspital Zürich - Discussing personalised mRNA vaccines and their CMC challenges. 🔶 View the full 2025 program here: https://lnkd.in/ehP39Apf #RNALeaders #siRNA #RNAediting #mRNA #ncRNA

Join us TOMORROW at 10 AM EST for our LSX RNA Leaders webcast, “Oligonucleotide Therapeutics and CMC Strategies,” with Asymchem! Sign up now: https://lnkd.in/e9mmDKJp Our featured speaker will discuss how Asymchem can effectively produce oligonucleotides at any scale with the required quality and quantity, from preclinical to commercial phases. Key topics: - Approved oligonucleotides and their synthesis technologies - Oligonucleotide regulatory challenges - Phase appropriate work scope and raw material specifications - Oligonucleotide analysis challenges - Asymchem oligonucleotide CDMO service capability and capacity Click the link below to register! https://lnkd.in/e9mmDKJp 

🧬RNA Leaders Industry Update🧬   ⭐Rgenta Therapeutics Inc. has announced a strategic partnership with GSK to progress discovery and development of RNA-targeted small molecule splice inhibitors. 🔗https://lnkd.in/eE2JjFnH   ⭐ Theratechnologies Inc. and Ionis Pharmaceuticals, Inc. have entered an exclusive licensing agreement focused on the commercialisation and development of two RNA-targeted therapies - olezarsen and donidalorsen in Canada. 🔗https://lnkd.in/eJfxa3Qv   ⭐Arrakis Therapeutics announced first presentation of preclinical data for its lead RNA-targeted small molecule program, which demonstrates reversal of myotonia in a mouse model of myotonic dystrophy Type 1. 🔗https://lnkd.in/ehr-3jvj   ⭐Moderna have opened its commercial-scale end-to-end mRNA manufacturing facility in Victoria, with capacity to produce up to 100M vaccine doses each year for respiratory diseases, the facility is the first of its kind in the southern hemisphere. 🔗https://lnkd.in/ggnKYeWv   Join RNA Leaders Europe this spring to hear more exciting updates!   🎤Jenny van Asbeck-van der Wijst , Mercurna - Discussing strategies for tissue targeting of mRNA therapies for immunomodulation and inflammatory diseases   🎤Generoso Ianniciello, Anima Biotech - Sharing insights on optimizing mRNA targeting and characterisation   🎤Riccardo Panella, Resalis Therapeutics - Exploring the design and development of anti miR-22 ASO for obesity   🎤Attila Seyhan, PhD, Brown University - Delving into the trials and tribulations of microRNA therapeutics 🔶 View the full 2025 agenda here: https://lnkd.in/eA4EmcX8   #RNALeaders #mRNA #RNAtargetting #microRNA

Join us next Wednesday for our LSX RNA Leaders Webcast featuring Asymchem on December 11, 2024 at 10 AM EST! Sign up now: https://lnkd.in/e9mmDKJp Our featured speaker will discuss how Asymchem can effectively produce oligonucleotides at any scale with the required quality and quantity, from preclinical to commercial phases. Key topics: - Approved oligonucleotides and their synthesis technologies - Oligonucleotide regulatory challenges - Phase appropriate work scope and raw material specifications - Oligonucleotide analysis challenges - Asymchem oligonucleotide CDMO service capability and capacity Click the link below to register! https://lnkd.in/e9mmDKJp  

🧬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