作者: Schwestka, Marko ; Moradian, Hanieh ; Wang, Yadong ; Kaufmann, Jörg ; Keil, Oliver ; Radloff, Katrin ; Gutbier, Birgitt ; Gossen, Manfred ; Santel, Ansgar ; Witzenrath, Martin ; Ahrens, Katharina ; Kneller, Laura ; Fehring, Volker ; Giese, Klaus ; Dunne, Charlotte Maeve ; Tondera, Daniel ; Moga, Akanksha ; Gkionis, Leonidas

Dysfunction of endothelial cells (ECs) lining the inner surface of blood vessels are causative for a number of diseases. Hence, the ability to therapeutically modulate gene expression within ECs is of high therapeutic value in treating diseases such as those associated with lung edema. mRNAs formulated with lipid nanoparticles (LNPs) have emerged as a new drug modality to induce transient protein expression for modulating disease-relevant signal transduction pathways. In the study presented here, we tested the effect of a novel synthetic, nucleoside-modified mRNA encoding COMP-Ang1 (mRNA-76) formulated into a cationic LNP on attenuating inflammation-induced vascular leakage. After intravenous injection, the respective mRNA was found to be delivered almost exclusively to the ECs of the lung, while sparing other vascular beds and bypassing the liver. The mode of action of mRNA-76, such as its activation of the Tie2 signal transduction pathway, was tested by pharmacological studies in vitro and in vivo in respective mouse models. mRNA-76 was found to prevent lung vascular leakage/lung edema as well as neutrophil infiltration in a lipopolysaccharide-challenging model.

Drug Development & Delivery

Platform technology the PTXΔLNP platform: on the promise of developing new LNPs for tomorrow's mRNA therapies

作者: Radloff, Katrin ; Gkionis, Leonidas ; Dunne, Charlotte

Molecular Therapy-Nucleic Acids

Cationic LNP-formulated mRNA expressing Tie2-agonist in the lung endothelium prevents pulmonary vascular leakage

作者: Wang, Yadong ; Schwestka, Marko ; Moradian, Hanieh ; Keil, Oliver ; Radloff, Katrin ; Gutbier, Birgitt ; Gossen, Manfred ; Kaufmann, Joerg ; Santel, Ansgar ; Witzenrath, Martin ; Kneller, Laura ; Ahrens, Katharina ; Fehring, Volker ; Dunne, Charlotte Maeve ; Tondera, Daniel ; Giese, Klaus ; Moga, Akanksha ; Gkionis, Leonidas

Dysfunction of endothelial cells (ECs) lining the inner surface of blood vessels are causative for a number of diseases.Hence, the ability to therapeutically modulate gene expression within ECs is of high therapeutic value in treating diseases such as those associated with lung edema. mRNAs formulated with lipid nanoparticles (LNPs) have emerged as a new drug modality to induce transient protein expression for modulating disease-relevant signal transduction pathways.In the study presented here, we tested the effect of a novel synthetic, nucleoside-modified mRNA encoding COMP-Ang1 (mRNA-76) formulated into a cationic LNP on attenuating inflammation-induced vascular leakage.After i.v. injection, the resp. mRNA was found to be delivered almost exclusively to the ECs of the lung, while sparing other vascular beds and bypassing the liver.The mode of action of mRNA-76, such as its activation of the Tie2 signal transduction pathway, was tested by pharmacol. studies in vitro and in vivo in resp. mouse models. mRNA-76 was found to prevent lung vascular leakage/lung edema as well as neutrophil infiltration in a lipopolysaccharide-challenging model.

项与 Pantherna Therapeutics GmbH 相关的新闻（医药）

2024-10-25

·pantherna-therapeutics.com

Pantherna Therapeutics and TQ Therapeutics announce research collaboration to advance ultra-short Cell and Gene Therapy (CGT) process leveraging LNP technology

PRESS RELEASE Pantherna Therapeutics GmbH and TQ Therapeutics GmbH announce a research collaboration to advance both of their proprietary platform technologies to include LNPs into an ultra-short extracorporeal cell therapy process. Pantherna Therapeutics GmbH, an mRNA-lipid nanoparticle (LNP) platform company dedicated to the research and development of innovative mRNA-based therapeutics team up with TQ Therapeutics GmbH, an advanced CGT company developing an ultra-short extracorporeal point-of-care process. The joint goal is to investigate novel strategies for combining both platforms on enhanced therapeutic cell generation. The efforts focus on exploiting the individual platform advantages regarding flexibility, safety and efficacy to obtain fast and cost-effective cellular therapies. Both companies believe combining both point of care process with LNP technology will have a major impact in solving the current challenges in cell-and-gene therapy to make cell therapies scalable and broadly accessible for many patients. “A collaboration of visionary teams is the cornerstone of innovation. In Pantherna Therapeutics, TQx has found a partner with whom we believe we can revolutionize cell therapies as they exist today. By uniting our technologies, we are creating a new platform for the production of CAR-T cells, making it accessible to everyone.” – Dr. Christian Eckert (CEO – TQ Therapeutics) “Pantherna is pleased to collaborate with TQ Therapeutics on their novel extracorporeal process for the generation of autologous CAR-T cell therapies. Our proprietary PTX-LNPs will serve as a non-viral delivery vehicle for this innovative approach. Together, we aim to significantly advance cell therapy and provide new treatment options for patients.” – Dr. Ansgar Santel (CEO – Pantherna Therapeutics) Pantherna Therapeutics company summary: Pantherna Therapeutics GmbH develops novel first-in-class therapies based on mRNA for indications with high medical need. Pantherna’s therapeutic programs are based on two innovative and proprietary platform technologies, the mRNA expression constructs (PTXmRNA®) and necessary lipid nanoparticles (PTXΔLNP®) for cell type-specific expression of therapeutic proteins. Pantherna is pursuing the development of a pipeline of novel mRNA-LNP candidates that will enable the therapeutic use of mRNA in various tissues and cell types. www.pantherna-therapeutics.com TQ Therapeutics company summary: TQ Therapeutics is a biotechnology company dedicated to advancing the field of cellular therapies through innovative technologies. The company’s mission is to develop a transformative cell processing platform, enabling advanced and affordable treatments in CGT. This technology will deliver highly individualized cell therapies directly to the patient’s bed-side. www.tqtherapeutics.com

细胞疗法

2024-09-26

·pantherna-therapeutics

Pantherna Announces Research collaboration with Bayer to Advance LNP Technology for Beyond Liver mRNA Therapy

Pantherna Therapeutics GmbH, a mRNA-lipid nanoparticle (LNP) platform company dedicated to the research and development of innovative mRNA-based therapeutics today announced a research collaboration with Bayer, investigating novel LNP-carriers for future mRNA therapies outside the liver. By combining their expertise in RNA therapeutics, both parties intend to identify novel LNP formulations that surpass the performance of conventional neutral LNPs as used in the mRNA vaccines. These advanced LNPs will be designed to potentially offer superior properties, ensuring the desired pharmacological outcomes and facilitating a smooth scalable translation towards development. Ansgar Santel, CEO of Pantherna Therapeutics, commented “Pantherna values Bayer’s trust in our technology and expertise. In collaboration, we will explore the potential of novel LNP formulations tailored to Bayer’s therapeutic interest, accelerating the pace of innovation and enhancing patient care. We look forward to partnering with the Bayer Non-Viral Delivery team.” Contact

信使RNA疫苗siRNA

2024-05-03

·pantherna-therapeutics

Company update: Ansgar Santel appointed as new CEO - Pantherna Therapeutics

Hennigsdorf, May 3, 2024 As of today, Ansgar Santel acts as the new Chief Executive Officer and becomes an additional member of the Board of Managing Directors at Pantherna Therapeutics GmbH with Jörg Kaufmann and Gerrit Maass. Ansgar joined Pantherna in November 2021 as Head of Translational Research and Alliance Management. He has a wealth of experience in developing new innovative RNA therapies whilst exhibiting exceptional leadership qualities. We at Pantherna are proud to welcome Ansgar into his new role as CEO and are confident in his commitment to accelerating Pantherna’s innovation and success in the development of impactful RNA therapies. Contact for additional information:   Dr. Ansgar Santel, CEO T: +49(0)3302-202 240-0  E-Mail: a.santel@pantherna-therapeutics.com  

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