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𝐏𝐥𝐚𝐬𝐦𝐢𝐝 𝐃𝐍𝐀 𝐂𝐨𝐧𝐭𝐫𝐚𝐜𝐭 𝐌𝐚𝐧𝐮𝐟𝐚𝐜𝐭𝐮𝐫𝐢𝐧𝐠 : 𝐀 𝐂𝐨𝐦𝐩𝐫𝐞𝐡𝐞𝐧𝐬𝐢𝐯𝐞 𝐎𝐯𝐞𝐫𝐯𝐢𝐞𝐰(𝐞-𝐏𝐃𝐅 𝐆𝐮𝐢𝐝𝐞) 𝐆𝐞𝐭 𝐚 𝐒𝐚𝐦𝐩𝐥𝐞 𝐞-𝐏𝐃𝐅 𝐨𝐟 𝐫𝐞𝐩𝐨𝐫𝐭 — https://lnkd.in/dP3BbSD7 The global #plasmid #DNA contract manufacturing market size is expected to reach USD 1,117.5 million by 2030, registering a CAGR of 19.3%, according to a new report by Grand View Research, Inc. The growing popularity of cell & gene therapy, prevalence of chronic diseases is increasing and plasmid DNA has a wide range of uses in the healthcare sector are the key factors driving the growth of the market. #gvr #marketresearch #globalmarket #marketindustry #research

#AAVdownstream challenges: expert insights #AAVvectors are a primary delivery mechanism for #genetherapies due to low immunogenicity, long lasting gene expression, and the ability to target specific tissues. The field of #AAV manufacturing is rapidly expanding, but there are challenges in the downstream processing of these #viralvectors. Next month, an expert panel including Nathalie Clément (VP, Vector Development for Translational Gene Therapies, Siren Biotechnology), Matt Roach (Director of AAV Production, BridgeBio), Nicolas Laroudie (Senior Field Application Scientist, Thermo Fisher Scientific), and William Kish (Principal Scientist - Downstream Process Sciences Engineering, Taysha Gene Therapies) will provide an opportunity to learn more about the latest trends and issues in #AAVmanufacturing, and specifically, how to address the most common downstream processing challenges Register for this #webinar below:

Today is National DNA Day, a day to celebrate the groundbreaking discovery of the double helix in 1953 and the successful completion of the Human Genome Project in 2003. On days like today, we are reminded of the important research and discovery that have resulted in two of the greatest scientific advancements of all time, both of which have accelerated advancements in #GeneTherapy and remain a significant driver of progress and #innovation to date. At Purespring, we have built on these powerful discoveries and are proud to be the first company to treat kidney diseases by directly targeting the podocyte with AAV gene therapy. Learn more about our novel approach here: https://lnkd.in/d8_G__CR #KidneyDisease #DNADay #Biotech #CellAndGeneTherapy

🧬Research on #EpigeneticEditing by #AngeloLombardo's team led to the development of a novel 'hit-and-run' platform based on transiently delivered engineered transcriptional repressors (ETRs) for precise gene silencing without DNA alteration. The recent breakthrough study published in #Nature represents a leap in gene therapy, demonstrating for the first time durable epigenetic silencing in vivo.       🔍 A crucial step in this process is pinpointing the exact locations to direct the ETRs on the target gene. The method set up by Lombardo's lab allows to meticulously screen ETRs in vitro, ensuring they offer stable, long-term silencing with minimal off-target effects.      🎥 Learn more about their ETR screening protocol in the JoVE Journal, presented by Alessandro Migliara and Martino Alfredo Cappelluti, PhD 👉https://lnkd.in/e_F_wY_3 

Prediction algorithms can tell you where they predict off target edits to take place. ResolveDNA will tell you where they actually take place and, how many cells you sequence have those off target edits.

Gene Therapy biomanufacturing can be challenging due to lack of process robustness, rigor and reproducibility. Complex, multi-parameter investigation of design space early in process development can help set the foundation for a robust bioprocess by identifying and optimizing key process parameters. Leveraging Culture Biosciences’ fleet of on-demand, cloud-connected bioreactors enables scientists to run large-scale, complex experiments with real-time data transparency. Download our App Note to learn about AAV upstream process development and optimization using Culture Biosciences' 250mL bioreactors to increase % full AAV capsids vital for your gene therapy. #processdevelopment #processoptimization #AAV #viralvector #genetherapy https://lnkd.in/e3n6uipQ

Interested in learning more about AAV viral vector production and optimization of bioprocesses critical for gene therapy projects, download the Culture Biosciences App Note. culturebiosciences.com #processdevelopment #processoptimization #aav #viralvectors #bioprocess

🔬 Research Alert! published in #DrugDevIndPharm 2022, VOL. 48, NO. 1, 1–11 🌟 A cationic cyclodextrin derivative-lipid hybrid nanoparticles for gene delivery effectively promotes stability and transfection efficiency 🌟 This research article explores the development of a novel gene delivery system using lipid-polymer hybrid nanoparticles (LHNPs) composed of α-cyclodextrin-conjugated generation-2 polyamidoamine dendrimers (CDG2) and lipids. The study demonstrates that these CDG2-LHNPs exhibit superior stability and transfection efficiency compared to traditional liposomes and polymers. The nanoparticles were stable for up to 15 days at various temperatures and showed high transfection efficiency mediated by multiple endocytosis pathways, particularly caveolae-mediated endocytosis, which helps avoid lysosomal degradation. The hybrid system's enhanced cell uptake and proton buffering capacity contribute to its effectiveness, making it a promising candidate for gene therapy applications. 📖 Read the full article here: https://lnkd.in/gcxwaYrs #GeneTherapy #Nanoparticles #DrugDelivery

Uncover the Potential of Mediphage Bioceuticals #msDNA for Gene Therapy Revolution! Mediphage's pioneering DNA vector, msDNA, holds the key to overcoming the persistent challenges in rAAV manufacturing. Our data reveals a remarkable increase in yield, accompanied by a significant rise in the percentage of full capsids. The impact of this breakthrough technology is truly transformative. Mediphage is poised to play a pivotal role in making rAAV more accessible to a wider patient population. With reduced manufacturing costs, we aim to fuel further research and studies, ultimately expanding the horizons of gene therapy. Contact us to learn more! #GeneTherapy #msDNA #Innovation #Mediphage

Prime editing, a novel way to edit genes that offers more functionality than CRISPR, is one step closer to human use. In a presentation at the annual European Society of Cell & Gene Therapy Congress in Brussels, Prime Medicine, Inc. announced that its prime editing technology was successfully used in primates to correct a rare liver gene mutation. The team said that although they will have to see clinical data before they can determine the clinical benefit in humans, “high-level prime editing in NHP is likely to support that we can achieve high-level editing in humans.” Learn more about this promising research here: https://hubs.li/Q02dHB9R0 #primemedicine #cellandgenetherapy #geneediting #liverdisease #biotech #cellandgenetherapy #CRISPR #liverdisease