CATUG

DNA vaccines consist of plasmid DNA (#pDNA) of bacterial origin which encodes for the target antigen that can elicit both humoral and cell-mediated #immunity. At the formulation level, DNA vaccines hold several benefits over whole pathogen (i.e., attenuated and inactivated viral vaccines) or protein-based vaccines, such as simple and rapid #manufacturing processes or the easy manipulation of the encoded antigen by DNA engineering. Additionally, the #thermostability of DNA vaccines tackles the complications linked to cold chain maintenance, which instead is essential for less stable vaccines (e.g., mRNA-based vaccines) to avoid the inactivation during supply. https://lnkd.in/gnsfCy6n #DNA #LNP #Lipid #Engineering

Tunable #LNP surface chemistries may enable efficacious delivery across a range of organs and cell types. Here is describe a method to electrostatically adsorb bioactive #polyelectrolytes onto LNPs to create layered LNPs (LLNPs). https://lnkd.in/eXNHXWuQ #mRNA #DNA #Uptake #Trafficking #Transfection #Treatment #Genetherapy #Engineering

Simultaneous and comprehensive #characterization of mRNA-loaded #LNPs (mRNA-LNPs) from multi-attribute analytical methods, preferably with the resolution of differentiating potentially heterogeneous particle populations, would represent a significant advance in the #analytical testing of mRNA-LNPs, thus facilitating efficient and robust formulation development. https://lnkd.in/exxbJjBe #Drugdevelopment #Development #Engineering #mRNA #LNP

✨ 𝑭𝒊𝒏𝒂𝒍 𝑫𝒂𝒚 𝒂𝒕 𝑬𝑺𝑮𝑪𝑻 𝟐𝟎𝟐𝟒 ✨ As this European Society of Gene and Cell Therapy annual conference draws to a close, we’re reflecting on an incredible week in Rome filled with team-building activities, stimulating discussions, and inspiring connections shaping the future of medicine. Congratulations to Dr. Paula Cannon from #USC, who inspired us with her work on 𝐞𝐧𝐠𝐢𝐧𝐞𝐞𝐫𝐢𝐧𝐠 𝐁 𝐜𝐞𝐥𝐥𝐬 𝐟𝐨𝐫 𝐜𝐮𝐬𝐭𝐨𝐦 𝐚𝐧𝐭𝐢𝐛𝐨𝐝𝐲 𝐩𝐫𝐨𝐝𝐮𝐜𝐭𝐢𝐨𝐧—a novel platform to both engineer Ab ligands and generate biomolecule-secreting plasma B cells. Later, in the 𝐍𝐨𝐧-𝐕𝐢𝐫𝐚𝐥 𝐕𝐞𝐜𝐭𝐨𝐫𝐬 / 𝐍𝐚𝐧𝐨𝐭𝐞𝐜𝐡𝐧𝐨𝐥𝐨𝐠𝐲 session, Dr. Raymond Schiffelers from Utrecht Uni gave a talk on "nucleic acid nanomedicine production at the bedside", bringing us closer to real-time, personalized treatment options. We also want to recognize Dr. Hadi Valadi from UniGothenburg for his presentation on "extracellular vesicles (#EVs) for RNA transport and therapeutic applications", a promising platform expanding current delivery options. Finally, we’d like to take this chance to thank all scientists, students, entrepreneurs and passionate people who stopped by our booth. We are here to make an impact, one groundbreaking project at a time. Thank you, for an unforgettable experience. We can’t wait to see what we will achieve together! 🌍💫 Lin Jin Chloe Wu Cesare Di Nitto #ESGCT2024 #GeneTherapy #CellTherapy #FutureOfMedicine #InnovationInScience #BiomedicalResearch #NonViralVectors #Nanotechnology #ExtracellularVesicles #RNAtherapeutics #PersonalizedMedicine #BiotechCommunity #ScientificImpact #MedicalInnovation #Rome

The long-term or excessive use of #Bisphosphonates (#BPs) typically leads to adverse effects such as gastrointestinal issues, decreased blood supply to bones resulting in increased fracture risks, and renal problems. There remains a need for rationally designed BP-modified ionizable #lipids that improve stability and mitigate potential side effects. A novel form of piperazine-based BP ionizable lipids was successfully developed using a robust piperazine ring structure (#PIP) conjugated to a #BP group. https://lnkd.in/e3TdKKnJ #LNP #Lipid #mRNA #Drugdelivery #Ionizable

Day 3 of #ESGCT2024 has been another exciting day! A morning highlight was Dan Peer's fascinating talk on cell-specific RNA delivery using #LNPs for vaccines and genome editing. Congratulations to Chiara Bonini on receiving the Outstanding Achievement Award for her inspiring work on T-cell engineering in oncology and autoimmunity. After lunch we could dive into the poster session. Congrats to Gabriele Alessandro Fontana who presented Cellvie's platform leveraging on mitochondria as delivery systems, and Laure Harinck from #UniGhent with her work on ex-vivo generation of CAR-T cells using mRNA-LNPs. We concluded the day with compelling talks by Chiara F Magnani and Sara Ghorashian, who presented highly promising clinical data that demonstrated significant progress in overcoming CAR-T therapy limitations in both AML and ALL patients. If you haven’t yet, stop by booth D34 you have one day left! #ESGCT2024 #GeneTherapy #CART #AML #ALL #Hematology #NonViralVectors #SleepingBeauty #MitochondrialTherapy #Innovation #Immunotherapy #CellTherapy #CancerResearch #Networking #CRISPR #GeneEditing #RareDiseases

Modified messenger #RNA (#modRNA) presents compelling properties that could allow new therapeutic strategies to tackle the underlying molecular pathways that ultimately lead to development of chronic heart failure. However, clinical application of modRNA for the heart is challenged by the lack of effective and safe delivery systems. During this study it was also determined both in vitro and in vivo that the cardiac cells targeted by modRNA-LNPs include #fibroblasts, #endothelial cells and #epicardial cells, suggesting that these cell types could represent targets for therapeutic interference with these LNP formulations. https://lnkd.in/e4c_6_rC #Cardiac #Heart #mRNA #LNPs #Lipid #Biodistribution #Drugadministration #Drugformulation #Engineering

Day 2 of ESGCT 2024 has been filled with inspiring talks, just as we expected! The morning plenary on AAV vectors in gene therapy for rare diseases offered a deep dive into cutting-edge solutions, with clinical results showing great progress in efficacy and safety profile. The Cancer Gene Therapy session was a highlight, particularly Chiara Quintarelli's talk on the challenges and opportunities in treating #pediatric #cancers with CAR-T therapies. In the Non-Viral Vectors/Nanotechnology I session, shoutout to @Jacek Lubelski for his presentation on using tLNPs to deliver DNA to T-cells, generating long-acting CAR-T cells in vivo by Nanocell Therapeutics! Thomas Hansen also impressed us with presenting circVec optimization, a circular RNA expression platform for enhanced gene therapy by Circio Holding ASA . If you haven’t stopped by booth D34 yet, come visit us! We’d love to connect and discuss your nucleic acid-based and GCT projects. CATUG Chloe Wu Cesare Di Nitto Lin Jin #ESGCT2024 #GeneTherapy #AAVVectors #CART #CancerResearch #PediatricCancer #NonViralVectors #Nanotechnology #tLNPs #GeneEditing #BaseEditing #Organoids #RegenerativeMedicine #CRISPR #Innovation #RNA

The world's first #mRNA #vaccine to treat #norovirus has started in the #UK. https://lnkd.in/eBm4W4qg

Starting the day with some BIG NEWS for CAR-T! Interius BioTherapeutics has dosed its first patient with INT2104, a gene therapy that delivers a CAR transgene to generate CAR-T/CAR-NK cells in vivo. I'd like to congratulate Philip R. Johnson, MD, Tim Culp, and the whole Interius team on this monumental achievement. If you missed it, I implore you to read about my conversation with Phil and Tim on Cell & Gene. https://lnkd.in/gFt9xv4e