Podcasts about pdna

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The first quarter earnings calls of major pharmaceutical companies were dominated by uncertainty surrounding President Trump's tariffs. Merck took a $200 million hit due to the tariffs, while Roche believes it is well-positioned to weather the impact. Despite challenges, companies are preparing for potential impacts of the tariffs on their businesses. This uncertainty is also affecting M&A activity in the pharmaceutical industry. Wacker Biotech is positioning itself as a partner for advanced therapies, offering services for process development and production of PDNA and mRNA therapies. Novavax asserts that its COVID-19 shot is "approvable", Vanda criticizes the FDA for delays, and there are updates on investments in innovative therapies. The industry is also reshaping the women's health landscape with economic challenges and strategic investments. Overall, companies are navigating through challenges while striving to thrive in a changing biotech landscape.

Wacker Chemie AG Company Presentation: Key Takeaways Introduction to Wacker ChemieJoerg Hoffmann, Head of Investor Relations at Wacker Chemie AG, presented the company's business model, core segments, markets, and strategies. Wacker Chemie, a leader in specialty chemicals and advanced materials, reported €6.4 billion in sales and €824 million in EBITDA for 2023, employing over 16,000 people globally. The company's innovation, sustainability, and leadership across its four business segments—Silicones, Polymers, Polysilicon, and Biosolutions—were emphasized. Wacker's technologies enable advances in modern computing, artificial intelligence, and renewable energy. The company consistently ranks #1 or #2 globally in its segments, leveraging vertically integrated operations and global reach for competitive advantage. Business Segments Overview SiliconesWacker's largest segment contributes significantly to revenue, with 85% of silicone sales from high-margin specialities. Silicones are used in industries such as construction, healthcare, automotive, and renewable energy. Applications include adhesives, insulation, e-mobility components, and coatings. Wacker's integrated production process ensures high-quality, specialized products. PolymersThis segment focuses on sustainable, water-based binders and adhesives used in paints, construction materials, and food packaging. Key innovations include dispersible polymer powders (DPP) and vinyl acetate-ethylene (VAE)-based adhesives, addressing urbanization and green building trends by replacing plastics with environmentally friendly materials. PolysiliconWacker is a global leader in high-purity polysilicon for semiconductor and solar applications. Nearly half of all computer microchips use Wacker's polysilicon, which supports technologies like AI and renewable energy. The segment's focus on premium-quality products strengthens its leadership in semiconductor and solar markets. BiosolutionsThe fastest-growing segment, Biosolutions delivers advanced medicines, biopharmaceuticals, and nutraceuticals. It focuses on trends like personalized medicine with innovations such as mRNA and pDNA manufacturing. Wacker's proprietary technologies enable cost-efficient, scalable production for pharmaceutical partners. Sustainability and Integrated OperationsSustainability is core to Wacker's strategy, with a commitment to reducing CO2 emissions by 50% by 2030. Integrated production processes and renewable energy sources, such as hydropower, enhance efficiency. Over two-thirds of Wacker's portfolio addresses customer sustainability needs, such as water-based adhesives and CO2-neutral materials. The SustainaBalance® strategy focuses on creating products with low environmental footprints while enabling sustainable technologies. Applications include wind turbines, solar panels, and energy-efficient building solutions. Growth Drivers and Strategic Vision Wacker's strategy emphasizes value over volume, targeting €10 billion in sales by 2030, an EBITDA margin above 20%, and a ROCE of 2x the cost of capital. Growth drivers include urbanization, energy efficiency, and the shift to renewables. Innovation and customer-centricity are key priorities, supported by a global network of technical centers delivering localized solutions. ▶️ Other videos: Elevator Pitch: https://seat11a.com/investor-relations-elevator-pitch/ Company Presentation: https://seat11a.com/investor-relations-company-presentation/ Deep Dive Presentation: https://seat11a.com/investor-relations-deep-dive/ Financial Results Presentation: https://seat11a.com/investor-relations-financial-results/ ESG Presentation: https://seat11a.com/investor-relations-esg/ T&C This publication is for informational purposes only and does not constitute investment advice. By using this website, you agree to our terms and conditions outlined on www.seat11a.com/legal and www.seat11a.com/imprint.

Introduction to Wacker Chemie Joerg Hoffmann introduces Wacker Chemie, a global leader in speciality chemical manufacturing. With over 16,000 employees, the company achieved an impressive €6.4 billion in sales and €824 million EBITDA in 2023, a testament to its exceptional performance. The company operates through four distinct business segments, each vitally involved in delivering innovative solutions to diverse industries worldwide. These segments are Silicones, Polymers, Polysilicon, and biosolutions. Business Segments at a Glance Silicones The largest segment, Silicones, represents a cornerstone of Wacker's business. Silicones are versatile materials in countless applications, from construction and healthcare to renewable energy and automotive. With 85% of sales driven by speciality silicone products, the segment demonstrates Wacker's focus on high-margin, customised solutions. The presentation also highlights the integration of silicones with polysilicon production, maximising resource efficiency. Polymers The Polymers segment specialises in adhesives, paints, and coatings, playing a key role in sustainable construction. Through innovations like dispersible polymer powders and vinyl acetate ethylene-based adhesives, Wacker delivers solutions that enhance energy efficiency and reduce carbon footprints. Applications include construction materials, food packaging, and textiles, with significant growth driven by the global trend toward urbanisation and green building standards. Polysilicon Wacker is the global leader in polysilicon manufacturing for semiconductor and solar applications. Nearly half of all computer microchips globally contain Wacker polysilicon, pivotal in enabling cutting-edge technologies such as artificial intelligence, connectivity, and renewable energy solutions. The solar segment also benefits from Wacker's superior quality and cost efficiency, which positions the company as a leader in sustainable energy transformation. Biosolutions The Biosolutions division is a fast-growing business focusing on biotechnology and advanced medicine. Through mRNA and pDNA production innovations, Wacker addresses high-growth markets in biopharmaceuticals and nutraceuticals. Its expertise in microbial fermentation supports next-generation pharmaceuticals, emphasising sustainability and scalability in drug manufacturing processes. Strategic Focus and Market Leadership Wacker Chemie prides itself on being a market leader in all its segments, ranking as #1 or #2 globally. The presentation underscores the company's unique position in leveraging its integrated value chain and advanced technology to provide tailored solutions across multiple industries, including construction, healthcare, electronics, and renewable energy. Wacker's commitment to innovation is evident in its continuous development of new products and solutions. ▶️ Other videos: Elevator Pitch: https://seat11a.com/investor-relations-elevator-pitch/ Company Presentation: https://seat11a.com/investor-relations-company-presentation/ Deep Dive Presentation: https://seat11a.com/investor-relations-deep-dive/ Financial Results Presentation: https://seat11a.com/investor-relations-financial-results/ ESG Presentation: https://seat11a.com/investor-relations-esg/ T&C This publication is for informational purposes only and does not constitute investment advice. By using this website, you agree to our terms and conditions outlined on www.seat11a.com/legal and www.seat11a.com/imprint.

While some of us knew a good bit about mRNA prior to 2020, we all got a crash course on mRNA technology and its prophylactic and therapeutic potential as a result of the COVID pandemic and subsequent SARS CoV-2 vaccine development. In fact, most of us have now received at least one mRNA vaccine at this point. Our guest for this episode, Dr. Christian Cobaugh, Co-founder and CEO of Vernal Biosciences, was a passionate believer in mRNA medicines well before the pandemic. Join us to hear his story and his passion for this technology. He walks us through the molecular methods by which high-purity mRNAs are now made and purified, as well as going into the lipid nanoparticle technology by which they're commonly delivered. As a contract development and manufacturing provider, we get to learn about the state of the market and what clients of their care about today. As a seasoned expert in this space, Christian talks about the future potential of mRNA technology for applications such as personalized cancer vaccines. If you enjoy hearing smart people talk about interesting topics with a passion, you won't want to miss this episode! Subscribe to get future episodes as they drop and if you like what you're hearing we hope you'll share a review or recommend the series to a colleague.  Download Transcripts: Speaking of Mol Bio Podcast | Thermo Fisher Scientific - US Visit the Invitrogen School of Molecular Biology to access helpful molecular biology resources and educational content, and please share this resource with anyone you know working in molecular biology.

Using cytokines therapeutically is like firing a shotgun at range – for sure you will hit your target, but off-target effects are likely, if not certain. To hit your target exclusively you have to get close. Delivering a proprietary version of the chronic inflammatory cytokine IL-10 encoded on a proprietary plasmid, injected locally, gets you really close. Using their non-viral, non-integrating vector, Xalud Therapeutics is exploring the use of IL-10 for osteoarthritis (FDA fast tracked), and other chronic inflammatory conditions, including ALS, MS, and neuropathic pain, with data from over 300 OA patients already in hand.

New vectors for successful transgene delivery in patients are more than needed. Current gene therapeutic vectors are mostly based on virus genomes. A main reason is the very efficient administration of the vector into the target cells. The viral background of these systems also has severe side effects like e.g. immunological reactions of the patient or the activation of oncogenes due to insertional mutagenesis, which can result in cancer development. A possibility to overcome these problems is the establishment of extrachromosomally maintained, autonomously replicating and non-viral vectors. The goal of this work was to establish such a novel system with the help of the CENH3 protein. The centromere-specific histone 3 variant CENH3 (H.sapiens: CENH3CENP-A, D.melanogaster: CENH3CID) is sufficient for centromere formation. It confers a stable and epigenetically heritable mark at the centromeric region and does this also on plasmids. With the help of CENH3 I changed the passive piggyback segregation mechanism of Epstein-Barr virus-derived vectors into an active segregation mechanism for plasmid DNA (pDNA) vectors. I demonstrated that the new and active pDNA vector segregation mechanism prolongs pDNA vector retention in cells. The information gained from basic research might have great impact in the field of gene-therapeutic research, as this mechanism might be a helpful tool in future gene therapeutic vectors.

Wed, 25 Sep 2013 12:00:00 +0100 https://edoc.ub.uni-muenchen.de/16146/ https://edoc.ub.uni-muenchen.de/16146/1/Noga_Matthaeus.pdf Noga, Matthäus ddc:540, ddc:500, Fakultät für

Tue, 4 Jun 2013 12:00:00 +0100 https://edoc.ub.uni-muenchen.de/15838/ https://edoc.ub.uni-muenchen.de/15838/1/Salcher_Edith.pdf Salcher, Edith ddc:540, ddc:500, Fakultät für Chemie und Pharmazi

The in vitro proliferation of prokaryotic and eukaryotic cells is remarkably hampered in the presence of heavy water (D2O). Impairment of gene expression at the transcription or translation level can be the base for this effect. However, insights into the underlying mechanisms are lacking. Here, we employ a cell-free expression system for the quantitative analysis of the effect of increasing percentages of D2O on the kinetics of in-vitro GFP expression. Experiments are designed to discriminate the rates of transcription, translation, and protein folding using pDNA and mRNA vectors, respectively. We find that D2O significantly stimulates GFP expression at the transcription level but acts as a suppressor at translation and maturation (folding) in a linear dose-dependent manner. At a D2O concentration of 60%, the GFP expression rate was reduced to 40% of an undisturbed sample. We observed a similar inhibition of GFP expression by D2O in a recombinant Escherichia coli strain, although the inhibitory effect is less pronounced. These results demonstrate the suitability of cell-free systems for quantifying the impact of heavy water on gene expression and establish a platform to further assess the potential therapeutic use of heavy water as antiproliferative agent.

The objective of the thesis was the lyophilization of cationic polymer-based pDNA or siRNA nanoparticles (i.e. polyplexes) with a special focus on the development of long-term stable formulations, the investigation of stabilization mechanisms especially during freezing, and the advanced monitoring of the lyophilization process. It was shown that an up-scaled preparation method in combination with subsequent lyophilization is a promising approach to reproducibly achieve long-term stable pDNA or siRNA polyplexes maintaining particle size and biological activity at pharmaceutically defined high quality. The results demonstrated that formulation development, highly dependent on the used type of polymer or nucleic acid, and process development, with a strong focus on the freezing, in combination with appropriate process monitoring needs always to be performed hand in hand. All in all, this thesis makes an essential contribution in order to move closer from a promising biotechnological approach towards clinic-friendly drugs.

In this work, we were able to take advantage of a deregulated wnt signaling pathway – a condition which is found in most gastrointestinal cancers, in particular in colorectal carcinomas. In order to restrict reporter gene expression to the desired cell type, we utilized the β-catenin dependent CTP4-promoter to restrict the expression of Firefly Luciferase and enhanced green fluorescent fusion protein (EGFPLuc) to cell lines with deregulated wnt signaling including SW480, LS174T, HepG2, Coga2 and Coga12. Stable cell lines containing this CTP4-driven EGFPLuc construct were established with the help of a lentiviral vector to monitor wnt activity by transgene expression. With these stably transduced cell lines, we performed a therapeutic target screen via siRNA-mediated knock-down of a number of potentially therapeutic targets within the wnt pathway – osteoprotegerin (OPG), Traf2 and Nck-interacting kinase (TNIK), SRY-related HMG-box (Sox2), protease-activated receptor 1 (PAR-1), β-catenin and transcription factor 4 (TCF4). The in vitro screening system was utilized as a prevalidation tool for therapeutically relevant targets. The degree of interference of our novel targets was determined and the search for a suitable siRNA target in colorectal cancer cells was narrowed down to β-catenin, PAR-1 and TNIK. As proof of principle the siRNA-mediated knock down of β-catenin was verified on mRNA and protein level in LS174T cells. After the initial read-out of various cell lines with different siRNAs has been established via the reduction of Luciferase expression levels, the biological effect of these targets were validated. For this purpose colony formation and cell motility/invasion assays were conducted for all relevant target cell lines. Furthermore in the in vitro experiments, the tumor-selectivtiy of the CTP4-promoter was employed in the delivery of the cytotoxic protein diphteria toxin A (DTA) in colorectal cancer target cells. Data evaluation of all in vitro assays pointed at reduced levels of proliferation, invasive behavior and aggressiveness, which yielded three candidates (PAR-1, TNIK and β-catenin) considered as viable for a treatment attempt in vivo. In the in vivo experiments, systemic delivery of siRNA against β-catenin, sticky siRNA targeting PAR-1 and plasmid DNA encoding for CTP4 controlled DTA were evaluated in a disseminated liver metastasis model of LS174T colorectal cancer. Specific knock-downs of β-catenin and PAR-1 were achieved which was confirmed via mRNA analysis. As for CTP4-DTA pDNA delivery the overall tumor load of the liver was reduced without any significant systemic toxicity, indicating specific DTA expression in tumor tissue. Also knock down of PAR1 using sticky siRNA significantly reduced tumor growth. All in all, the therapeutic effect of PAR-1 and β-catenin knock-down could be verified in various in vitro assays analyzing invasive behavior and anchorage independent growth and ultimately also in vivo. The tumor-specific expression of DTA pDNA could also be confirmed in vitro and was further investigated in an orthotopic liver dissemination model in NMRI nude mice.

In der vorliegenden Arbeit wurden A549 Zellen, als Modell für Epithelzellen der Lunge, mittels nicht-viralen Gentransfers mit pDNA bzw. mRNA kodierend für das sekretorische Protein mSEAP transfiziert. Die höchste Transgen-Expression konnte durch den mRNA-vermittelten Gentransfer erreicht werden, unabhängig von dem verwendeten Genvektor. Die Messung der Aktivität von mSEAP im Medium nach mRNA Transfektion mit Liposomen ergab eine Konzentration von maximal 1 ng/50 µl nach 24h, 5 ng/50 µl nach 48h und 7,5 ng/50 µl nach 72h. Für die pDNA Transfektion ergaben sich Konzentrationen für mSEAP von 400 pg/50µl nach 24 h, 700 pg/50 µl nach 48 h und 800pg/50 µl nach 72 h. Es konnte gezeigt werden, dass es bei der Verwendung von mRNA - vor allem nach Transfektion mit dem kationischen Lipid DMRIE-C - zu einer 9-fachen Steigerung der Proteinsekretion in das Medium der kultivierten Zellen kommt. Weiterhin waren ca. 45% der Zellen bei Verwendung von Lipoplexen mit DMRIE-C/mRNA im Gegensatz zu 15% nach Transfektion von pDNA positiv für mSEAP. Nach Transfektion der mRNA mit dem kationische Polymer b-PEI konnten Konzentrationen von mSEAP im Medium von 10pg, 15pg und 50pg in 50µl Medium nach 24h, 48h und 72h gemessen werden. Für die Magnetofektion von mRNA ergeben sich Mengen von ca. 150pg, 250pg und 400pg in 50µl Medium nach 24h, 48h und 72h. Dies entspricht im Vergleich zur Verwendung von pDNA einer 3,3-fachen Steigerung nach Transfektion von mRNA/b-PEI Komplexen, mit der Methode der Magnetofektion einer 4-fachen Steigerung der Proteinexpression. Die erhöhte Proteinexpression kann auf die intrazelluläre Barriere des nukleären Imports von pDNA, welche bei der Verwendung von mRNA nicht notwendig ist, zurückgeführt werden. Die Unterschiede des endosomalen „Escape“ - und dem daraus resultierenden Verhältnis freier mRNA zu komplexierter mRNA im Zytoplasma - könnte eine Erklärung für die geringere Effizienz von Polyethyleniminen im Vergleich zu kationischen Lipiden bei dem Transfer von mRNA sein. Weiterhin konnte mit diesem Modell gezeigt werden, dass humane alveolare Epithelzellen des Adenokarzinoms (A549) - als Modell für humane Lungenepithelzellen - imstande sind, zellfremde Proteine nach Transfer genetischen Materials zu translatieren und zu sezernieren. Eine pulmonale Applikation von therapeutischen Genen kodierend für sekretorische Proteine mittels Vernebelung könnte als nicht-invasive Methode z.B. für die Therapie von Hämophilie A oder B eine Rolle spielen. Weiterführende in vivo Versuche könnten Aufschluss über Serumkonzentrationen des sekretorischen Proteins nach pulmonaler Applikation bringen. Die Verwendung von mRNA als therapeutischem genetischem Material zeigt hierbei Vorteile gegenüber von pDNA, aufgrund der gesteigerten Transgen -Expression, der verminderten Dosis an Transferreagenz, sowie dem fast gänzlich ausgeschlossenen Risiko der insertionellen Mutagenese.

Thu, 15 Jan 2009 12:00:00 +0100 https://edoc.ub.uni-muenchen.de/11108/ https://edoc.ub.uni-muenchen.de/11108/1/Meyer_Martin.pdf Meyer, Martin ddc:540, ddc:500, Fakultät für Chemie und Pharmazie

Modification of cellular functions by overexpression of genes is increasingly practised for research of signalling pathways, but restricted by limitations of low efficiency. We investigated whether the novel technique of magnetofection (MF) could enhance gene transfer to cultured primary endothelial cells. MF of human umbilical vein endothelial cells (HUVEC) increased transfection efficiency of a luciferase reporter gene up to 360-fold compared to various conventional transfection systems. In contrast, there was only an up to 1.6-fold increase in toxicity caused by MF suggesting that the advantages of MF outbalanced the increase in toxicity. MF efficiently increased transfection efficiency using several commercially available cationic lipid transfection reagents and polyethyleneimine (PEI). Using PEI, even confluent HUVEC could be efficiently transfected to express luciferase activity. Using a green fluorescent protein vector maximum percentages of transfected cells amounted up to 38.7% while PEI without MF resulted in only 1.3% transfected cells. Likewise, in porcine aortic endothelial cells MF increased expression of a luciferase or beta-galactosidase reporter, reaching an efficiency of 37.5% of cells. MF is an effective tool for pDNA transfection of endothelial cells allowing high efficiencies. It may be of great use for investigating protein function in cell culture experiments.