Raquel Mura’s Post

Five bits of biotech news that got me excited (or scared) this week:   Regeneron Pharmaceuticals have 'dramatically improved' the hearing of two children with genetic deafness. Both had defective copes of the otoferlin gene which the new treatment targets.  https://lnkd.in/eWHwfTFu A vaccine for glioblastoma is showing early promise in human trials. The technique includes an interesting way of inducing both the innate and adaptive immune systems to destroy the cancer. https://lnkd.in/e6qUZ8Fe   Google deep mind have released alphafold-3 which can model the structure of a protein while it interacts with other molecules. It’s a powerful tool for determining not only what a protein looks like, but how it works in the cell, and its getting good reviews from scientists. https://lnkd.in/egjC3CZm Astrazeneca had decided to halt production of its COVID vaccine as other pharmaceutical giants also see reductions in revenue. https://lnkd.in/ez-edwB2 The market COVID vaccines may be waning but increasing fears that dairy cows in the US are forming a reservoir for new bird flu strains might mean new opportunities are on the way… https://lnkd.in/eMxWRtJB  …   I write about biological complexity as well as trends in the biotech and pharma industries. Next week I will be focussing on how mRNA delivery technologies have changed the way we think about vaccination. 

📢 Join InDevR, Inc. for an informative webinar on the latest advancements in RNA-based vaccine product characterization! 💉 With the rapid growth in mRNA #VaccineDevelopment, robust methods for characterizing #mRNA constructs and gene products have struggled to keep pace. Randy Lacey will introduce a cutting-edge multiplexed imaging platform designed to address these evolving needs. 🔬 Discover how VaxArray technology is revolutionizing mRNA vaccine processing and characterization. ➡️ Learn more + register here: https://buff.ly/3zAzMiv #Vaccine #DrugDevelopment #RNA #Regulatory #LaboratoryTechnology #VaccineDevelopment #VaccineManufacturing #mRNAVaccines #mRNATherapeutics

𝐀𝐛𝐥𝐚𝐭𝐢𝐧𝐠 𝐭𝐡𝐞 𝐈𝐦𝐦𝐮𝐧𝐨𝐠𝐞𝐧𝐢𝐜𝐢𝐭𝐲 𝐨𝐟 𝐈𝐕𝐓 𝐦𝐑𝐍𝐀 🌟 Managing the immune response to in vitro transcribed (IVT) mRNA is crucial in the field of RNA therapeutics. Toll-like receptors (TLRs), expressed by innate immune cells, are pattern recognition receptors devoted to recognizing a distinct set of molecular patterns that are not found in healthy cells. -𝑻𝑳𝑹-𝟑 𝒄𝒂𝒏 𝒔𝒆𝒏𝒔𝒆 𝒅𝒐𝒖𝒃𝒍𝒆-𝒔𝒕𝒓𝒂𝒏𝒅𝒆𝒅 𝑹𝑵𝑨 (𝒅𝒔𝑹𝑵𝑨) 🧬 -𝑻𝑳𝑹-𝟕 𝒂𝒏𝒅 𝑻𝑳𝑹-𝟖 𝒔𝒆𝒏𝒔𝒆 𝒔𝒊𝒏𝒈𝒍𝒆-𝒔𝒕𝒓𝒂𝒏𝒅𝒆𝒅 𝑹𝑵𝑨 (𝒔𝒔𝑹𝑵𝑨) 〰️〰️ When activated, TLR3, TLR7, and TLR8 trigger transcription factors like IRF3, IRF7, and NF-κB, leading to the production of inflammatory cytokines (IL-6, IL-12, TNF-α) and type I interferons (IFN-α, IFN-β). 🔥 Research breakthroughs and optimized manufacturing processes have contributed to suppressing IVT RNA-induced immunogenicity mainly by: •𝐍𝐮𝐜𝐥𝐞𝐨𝐬𝐢𝐝𝐞 𝐌𝐨𝐝𝐢𝐟𝐢𝐜𝐚𝐭𝐢𝐨𝐧: Incorporation of methylated nucleosides and pseudouridine dampen TLR recognition, as shown by K. Karikó's work published in Immunity in August 2005. In addition, modified RNA is also less likely to form dsRNA structures, such as hairpins potentially triggering TLR3. •𝐏𝐮𝐫𝐢𝐟𝐢𝐜𝐚𝐭𝐢𝐨𝐧 𝐓𝐞𝐜𝐡𝐧𝐢𝐪𝐮𝐞𝐬: Using methods like reverse phase HPLC (RP-HPLC) can effectively remove dsRNA impurities. CATUG in-house technology has earned us strong reputation in removal of dsRNA contaminants dramatically reducing RNA-related immunogenicity while increasing treatment efficacy. 🤸♂️ Join us in advancing high purity RNA therapeutics! Reach out to me or my colleague Congyu (Chloe) Wu if you'd like want to know more. 😉 www.catug.bio/contact #RNAtherapeutics #Immunogenicity #Immunology #Biotechnology #InnateImmunity #TLRs #Cytokines #Interferons #NucleosideModification #RPHPLC #Immunity #RNAvaccine #mRNAtechnology #dsRNAremoval #Inflammation #BiotechInnovation #Therapeutics #AdvancedMedicine

Successfully completed the guided project on COVID 19 mRNA Vaccine Degradation Prediction provided by Coursera. This guided online project provided in-depth knowledge on the mechanisms of mRNA degradation, a crucial aspect in understanding the stability and efficacy of mRNA-based vaccines. Through this program, I gained valuable insights into the latest research and predictive models that can aid in the development of more effective vaccines and therapies. Looking forward to applying these learnings in future projects and contributing to advancements in the field of mRNA technology and infectious disease research! #COVID19 #mRNA #Vaccines #Research #LifelongLearning #Biotechnology #genetics

#nobelprize2023 winner, Drew Weissman, talks about the endless potential of #RNA therapeutics in the development of #vaccines and in the treatment of many #diseases! The future is bright in #scienceresearch & #healthcare!! #rnatherapeutics #mrnavaccines #healthandwellness #healthcareinnovations #healthandsocialcare #scienceandtechnology

As we continue to discover new treatment approaches, we need to go beyond targeting proteins with well-defined pockets and find other ways to target the undruggable. Two approaches that show promise are PROTACs and molecular glues, writes Stephen Fawell of AstraZeneca in this new article. https://lnkd.in/e2ju_Cbz #drugdiscovery #drugtargeting #protac

Rise of the RNA machines – Self-Amplification in mRNA Vaccine Design Replicons Opening Pandora's Box for Changing Populations Forever with a Flood of New Shots Comes et al, published an enthusiastic review on self-amplifying mRNA vaccines which takes concerns existing today with Pfizer and Moderna to whole new levels. Replicons in this manuscript are not Transformer action figures, they are self-replicating biosynthetic genetic vaccines designed to reproduce themselves in the human body. Read on Courageous Discourse: https://shorturl.at/CG6I5 #MFScholar

Lipid Nanoparticles (LNPs) are promising non-viral delivery vehicles composed of a specific mixture of lipids including cationic/ionizable lipids, and other excipients. A technology that has been in development for some time, the recent COVID-19 vaccines by companies such as Moderna and Pfizer have vaulted LNPs into the spotlight as critical delivery systems of nucleic acids, especially messenger RNA (mRNA). This technology not only protects the payload but also overcomes multiple physiological barriers. In addition to facilitating rapid vaccine development, LNPs are capable of carrying and delivering therapies that may be useful in a number of applications, including infectious disease, immuno-oncology, genetic disorders, and neurodegenerative conditions. In this application note, we explore how Microfluidizer® technology plays a vital role in safely producing lipid nanoparticles. Learn more about our case study with Cayman Chemical Company which uses Microfluidizer top-down technology to achieve small, uniform particle sizes for LNP production. https://hubs.la/Q02MC8GR0 #LNPs #topdownmanufacturing #lipidnanoparticles #LNPmanufacturing #vaccinemanufacturing #Microfluidics #Microfluidizertechnology #Microfluidizerprocessor

Have a look at the final version of the publication in JACS introducing the cap analog we called AvantCap. The structure, synthesis, IVT, and performance of mRNA in cell cultures and in vivo. All there. Enjoy! #mrna #mrnavaccines #mrnatherapeutics

So this discovery /does/ matter, a lot. This is just the tip of the iceberg, why you should want a DNA vaccine delivered by an adaptation of a natural process, over a translation-ready mRNA vaccine delivered by an exotic package. The DNA vaccine that your cells transcribe to mRNA themselves keeps your own body in a lot more control, and this research reveals just how deep that control goes. Raise your hand 🙋🏻♂️ if you felt like complete and utter garbage the day after each Pfizer shot; why should that be happening, when it never happens with a flu vaccine? https://lnkd.in/gYuEZ4if