Phospholipid Research Center Heidelberg’s Post

Grateful for the opportunity to attend the Agilent Biopharma Innovations Summit ! This event was an invaluable opportunity to delve into the latest advances across biopharmaceutical research and development. Key sessions included: 1. Genomics Applications for mRNA Workflows – Hosted by Dr. Hervé Chaulet, this session highlighted end-to-end solutions in IVT mRNA development, protein therapeutics, and gene therapies. 2. Quality Control in mRNA Vaccine Development – Led by Dr. Leslie Friedmann, this talk focused on the Agilent Fragment Analyzer systems' role in reliable, precise mRNA QC, covering checkpoints from plasmid quality to final product integrity. 3. Innovations in Monoclonal Antibody Testing – Dr. Kyle Luttgeharm and Steve Siembieda demonstrated the powerful capabilities of Agilent’s 12-channel CE-SDS ProteoAnalyzer, optimized for efficiency and cost-effectiveness in assessing critical quality attributes like glycosylation and monomeric purity. 4. Advances in Cell Analysis for Immune-Based Therapies – Dr. Kelly Kroeger explored tools enabling real-time cell function assessment, empowering researchers to unlock the potential of immune cell-based therapeutics. Agilent's advanced tools and methodologies continue to push the boundaries in biopharma, from early discovery to quality assurance. Excited to apply these insights to future projects! #Biopharma #DrugDiscovery #mRNA #Immunology #QualityControl #Innovation #Agilent

🔬 Endosomal Escape: Overcoming a Critical Challenge in LNP Therapeutics! 🚀 Get an in-depth understanding of this key obstacle in #LNP-mediated therapeutics by exploring endosomal escape - the process by which nanoparticles exit endosomes to release their cargo into the cell's cytoplasm. Our latest article dives into this crucial bottleneck in #mRNA #therapy development, including #vaccines and #gene editing, offering insights into: - Mechanisms driving #Endosomal Escape: Understanding how #nanoparticles navigate cellular pathways. - Endosomal escape optimizations: Discover the strategies to enhance endosomal escape efficiency. - Detection Methods: Find out more about the technics to monitor and measure escape processes. 🧬 Don't miss this in-depth review and join the conversation on advancing RNA delivery systems! 👉 Read more: https://lnkd.in/efdmAHJf

RNA therapeutics derived from mRNA and miRNA are emerging as pivotal tools against health crises by promising new, life-changing therapies. Adopt faster analytical solutions to characterize RNA: https://bit.ly/3BISMMe #DanaherLifeSciences #RNA #mRNA #miRNA #generegulation

https://lnkd.in/gCHvi2db In the endogenous regulatory process called RNA interference (RNAi), short double-stranded RNA oligonucleotides cause sequence-specific posttranscriptional silencing of genes. Small interference RNA (siRNA) has now emerged as a promising therapeutic strategy for the development of novel drugs. However, for successful systemic delivery of siRNA therapeutics, siRNAs must penetrate cellular barriers to reach their targets in the cytoplasm... Our Director of Scientific Operations, Dr. Klaus Linse does excellent research, learn more of his discoveries at biosyn.com. Klaus D. Linse, Ph.D.

1. The amount of RNA therapeutics approved for Phase 1 clinical trials globally is increasing at a steady rate; this is due to advancement in RNA technologies, interest in RNA therapeutics, innovations in drug delivery systems, and clear guidelines established for the development and approval of RNA therapeutics by the FDA and EMA. 2. Due to the above factors, the number in 2024 is set to eclipse that in 2023 by a substantial margin. 3. Lipid nanoparticles continue to be a popular choice of drug delivery system due to the success of COVID-19 mRNA vaccines (Note: a large portion of "Other" is undisclosed). 4. Most RNA therapeutic types are increasing in numbers. Interestingly, antisense oligonucleotides (ASOs), seen a dip shortly after the pandemic but have since maintained relevancy. 5. In 2024, guide RNA make up almost a third of RNA therapeutics approved for Phase 1 clinical trials, highlighting the rise of gene editing technologies such as CRISPR-Cas9 in treating genetic disorders, cancers, and infectious diseases. Data obtained from Beacon Intelligence using the "RNA therapeutics" and "Trial Start Date" parameters. #RNA #RNAtherapeutics #mRNA #mrnatherapeutics #mrnavaccines #LNP #lipidnanoparticles #liposome #mrnalnp #drugdelivery #crispr #crisprcas9 #geneediting #genetherapy #helixbiotech

This study evaluates the use of size-exclusion chromatography (SEC), multi-angle light scattering (MALS) detection, and mass photometry for characterizing messenger ribonucleic acid (mRNA). With the recent approval of mRNA vaccines for COVID-19, understanding mRNA quality is crucial for its broader applications in cancer immunotherapy, protein replacement therapy, and gene editing. The study explores the impact of SEC parameters on separating different forms of mRNA and introduces MALS and mass photometry for accurately determining mRNA molecular weight and studying its structure under native conditions. #mRNACharacterization #SizeExclusionChromatography #MultiAngleLightScattering #MassPhotometry #mRNAQuality #COVID19Vaccines #Biopharmaceuticals #GeneTherapy #ProteinTherapeutics #StructuralAnalysis

Chemically Modified Platforms for Better RNA Therapeutics:- •RNA-based therapies have catalyzed a revolutionary transformation in the biomedical landscape, offering unprecedented potential in disease prevention and treatment. •However, despite their remarkable achievements, these therapies encounter substantial challenges including low stability, susceptibility to degradation by nucleases, and a prominent negative charge, thereby hindering further development. •Chemically modified platforms have emerged as a strategic innovation, focusing on precise alterations either on the RNA moieties or their associated delivery vectors. •This comprehensive review delves into these platforms, underscoring their significance in augmenting the performance and translational prospects of RNA-based therapeutics. •It encompasses an in-depth analysis of various chemically modified delivery platforms that have been instrumental in propelling RNA therapeutics toward clinical utility. •Moreover, the review scrutinizes the rationale behind diverse chemical modification techniques aiming at optimizing the therapeutic efficacy of RNA molecules, thereby facilitating robust disease management. •Recent empirical studies corroborating the efficacy enhancement of RNA therapeutics through chemical modifications are highlighted. •Conclusively, we offer profound insights into the transformative impact of chemical modifications on RNA drugs and delineates prospective trajectories for their future development and clinical integration.

📢 𝑹𝑵𝑨 𝑬𝒅𝒊𝒕𝒊𝒏𝒈 𝒊𝒔 𝒎𝒂𝒌𝒊𝒏𝒈 𝒊𝒕𝒔 𝒘𝒂𝒚!! The first RNA Editing candidate #WVE006 from Wave Life Sciences gets the approval for Phase 1b/2a (RestorAATion-2 trial), after standardizing the starting dose in healthy controls. WVE-006 is an RNA Editing oligonucleotide designed to correct the homozygous Pi*ZZ mutation in 𝘚𝘌𝘙𝘗𝘐𝘕𝘈 1𝘡 allele (mRNA editing) leading to 𝐚𝐥𝐩𝐡𝐚-1 𝐚𝐧𝐭𝐢-𝐭𝐫𝐲𝐩𝐬𝐢𝐧 𝐝𝐞𝐟𝐢𝐜𝐢𝐞𝐧𝐜𝐲 (AATD) causing liver/lung disease or both. It is conjugated with Gal4NAc and is administered subcutaneously. This specific correction will restore the levels of functional AAT protein, which will be measured as the primary endpoint of this trial. Following the RestorAATion-2 trial, GSK will take over the development and commercialization of WVE-006. Am glad to be carrying out this technology at the preclinical level for a different disease and to see it gaining clinical momentum keep my hopes high 👍 #genetherapy #RNA https://lnkd.in/g9kNWDmv

📣 Slides available for download! 💻 Last week, Andreas Wagner from Polymun Scientific Immunbiologische Forschung GmbH gave a #webinar on the versatile #LNP production process from #mRNA vaccines, therapeutics, and gene editing. You missed this online #seminar? Slides of the presentation are now available for download! 👉 Check our website. #education #lipidnanoparticles #geneediting #vaccine #RNA

"Bile Acid-Containing Lipid Nanoparticles Enhance Extrahepatic mRNA Delivery" is a great #article highlighting how Bile Acids can be used to take #mRNA outside of the liver. ⚪ ⚫ 🔴 🔵 Through #microfluidic mixing, the scientists used various #Lipids and #BileAcids to optimize their formulation. "Here, we present a strategy for achieving extrahepatic delivery of mRNA involving the incorporation of bile acids, a naturally-occurring class of cholesterol analogs, during LNP synthesis. We synthesized a series of bile acid-containing C14-4 LNPs by replacing cholesterol with bile acids (cholic acid, chenodeoxycholic acid, deoxycholic acid, or lithocholic acid) at various ratios." The results show that a four-component BA-LNP formulation, CA-100, for extrahepatic mRNA delivery that could potentially be utilized for a range of therapeutic applications. Congratulations to Savan Patel, Margaret Billingsley, Alvin Mukalel, Ajay Thatte, Alex Hamilton, Ningqiang Gong, Rakan El-Mayta, Hannah Safford, Maria Merolle, and Michael J Mitchell for their great work! https://lnkd.in/efJcucSn #LNP #Excipients #BALNP #Research #Vaccines #VaccineResearch #Cancer #CancerResearch #Innovation #DrugDevelopment #Science #Biology #DDS #pubmed