Tag: lipid nanoparticles
Niel, Belgium, July 22, 2025 –  etherna immunotherapies NV (“etherna”), a biotech company and a platform technology leader in pioneering mRNA and Lipid Nanoparticle (LNP) technologies, today announces breakthrough innovation in the efficient in vivo extrahepatic delivery of mRNA to several key tissues, including to hematopoietic and progenitor stem cells (HSPCs) in bone marrow and to T cells. Key highlights of the data presented include:
- New rodent and non-human primate data on proprietary etherna vaccine LNPs that outperform by at least 5-fold the clinical benchmark LNP compounds for vaccine applications
- New rodent and non-human primate data using etherna liver LNP that outperform by at least 4-fold the clinical benchmark LNP compounds for liver repeat dose protein expression and gene editing applications
- New LNPs that have been optimized for efficient delivery of RNA payloads to hematopoietic stem and progenitor cells (HSPC) in the bone marrow, achieving mRNA payload expression in over 90% of all mouse bone marrow HSPCs using an untargeted LNP
- Other newly described and optimized LNPs that have shown efficient delivery to T cells in vivo in both mice and humanized mouse models, including demonstration of robust CAR-T mediated killing using both untargeted and CD8 antibody-targeted LNP
- Data to be presented by Antonin de Fougerolles at the 5th annual mRNA-based Therapeutics Summit held in Boston
Nucleic acid-based therapeutics, including mRNA-based medicines, are rapidly reshaping the treatment landscape for genetic diseases, vaccines, and protein replacement therapies. While lipid nanoparticles (LNPs) have proven highly effective in liver-targeted applications, achieving robust delivery to extrahepatic cells remain a major challenge.
etherna’s proprietary library of bio-reducible ionizable lipids has been key in addressing this delivery challenge. In etherna’s recent Non-Human Primate (NHP) studies, the company’s proprietary LNP formulations demonstrated at least 5-fold improved mRNA expression compared to clinical benchmarks for both vaccine and liver repeat-dose protein expression applications. These LNPs were also very well tolerated in NHP and this improvement in liver-directed expression was also demonstrated in a rodent transthyretin gene editing study where it significantly outperformed the LP01 clinical benchmark and where equivalent efficacy was seen at ¼ the LP01 dose. These results are further proof of the potency and translational potential of etherna’s proprietary lipid library.
etherna has now developed a Bio-Reducible LNP for delivery of mRNA to bone marrow stem cells, a novel LNP platform optimized for extra-hepatic delivery. This platform is systematically optimized for increased mRNA expression through proprietary linker, tail, and headgroup chemistry modifications.
We have optimized an LNP formulation specifically for hematopoietic stem and progenitor cells (HSPCs). This formulation enabled a controlled shift in biodistribution away from the liver and toward bone marrow tissue. The resulting formulations achieved mRNA expression in over 90% of all bone marrow HSPCs, marking a transformative leap in the ability to deliver RNA therapeutics systemically to extrahepatic cell types.
This breakthrough was made possible through a structure activity relationship (SAR) optimization process which significantly enhanced the targeting of hematopoietic stem cells (HSCs). By adjusting lipid composition and systematically analyzing biodistribution outcomes, the research team unlocked a delivery profile that favors bone marrow uptake while maintaining stability, safety, and high transfection efficiency.
In addition, using a similar but slightly different approach, etherna scientists have optimized other LNP formulations for improved delivery to T cells. Efficient delivery to T cells using a passive targeting approach (consisting of just an ionizable lipid, helper lipid, cholesterol, and PEG lipid) was demonstrated in rodents. The addition of an anti-CD8 antibody on the LNP surface served to further increase in vivo uptake and mRNA expression in CD8+ T cells.
Importantly, we also demonstrated the ability of both our non-targeted and targeted T cell LNP formulations to deliver mRNA in a humanized mouse (mice reconstituted with human immune cells). When we used these formulations to deliver a CD19-CAR mRNA, a dramatic CAR-T-mediated killing of B cells was seen with both our untargeted and targeted LNP formulations. The level of CAR-T expression and activity seen using an unoptimized CD19-CAR mRNA was comparable to the current pre-clinical state of the art in the field, and we expect this to improve a further 3-5 fold upon incorporation of etherna’s proprietary mRNA designs.
These innovations represent a major advancement in the field of in vivo liver gene editing and therapeutic protein expression in hematopoietic stem cells and T cells. etherna’s breakthrough research offers a new treatment possibilities for hematologic disorders and beyond.
These data will be presented on July 22nd 2025 at the 5th annual mRNA-based Therapeutics Summit in Boston. This innovation positions etherna as a leader in Bio-Reducible LNP for delivery of mRNA to bone marrow stem cells, addressing a key challenge in extrahepatic targeting.
We welcome partners who are committed to pushing the boundaries of what’s possible in nucleic acid-based medicine therapeutics and delivery of payload to extrahepatic cell types.
Contact information:
etherna: media@etherna.be
www.etherna.be/contact
Niel, Belgium and Boston, USA, January 9, 2025 – etherna immunotherapies NV (“etherna”), a technology platform leader pioneering mRNA and lipid-based nanoparticle (LNP) technologies, announces a strategic collaboration with Dropshot Therapeutics (“Dropshot”). The collaboration combines the cutting-edge platforms of both companies to accelerate the development of RNA-based therapeutics for the development of multiple new drug candidates across several indications. etherna will receive an upfront payment and research funding, and potentially milestone payments during development and tiered royalties following product launch, together estimated to be up to USD 950 million.
As a recognized leader in mRNA and LNP technology, etherna has developed a suite of proprietary lipid nanoparticle formulations. These technologies enable precise delivery and expression of RNA therapeutics in targeted organs and cells, forming the foundation for groundbreaking treatments across multiple therapeutic areas.
Dropshot, focused on addressing unmet medical needs in cardiac and renal diseases, has been at the forefront of developing novel therapies utilizing LNPs to deliver RNA payloads. The collaboration builds on Dropshot’s prior evaluation of etherna’s mRNA and LNP platforms and is intended to create novel proprietary therapeutics based on etherna’s mRNA and its LNP formulations to move rapidly from pre-clinical animal studies into clinical human studies.
“This agreement underscores etherna’s commitment to driving innovation through collaboration,” said Bernard Sagaert, CEO of etherna. “Dropshot’s expertise in heart and kidney disease aligns with etherna’s mission to be a technology leader in the development of nucleic acid-based medicines. etherna aims to empower our partners to create transformative therapeutics and improve patients’ lives.
Marijn Dekkers, CEO of Dropshot, said “At Dropshot we have been working with etherna for some time evaluating their technology solutions to meet our needs to develop new potential RNA therapeutics. We are excited about this strategic collaboration wtih Dropshot to bringing our expertise in cardiac and renal disease biology together with etherna’s mRNA and LNP technology. We look forward to bringing multiple new drug candidates into active development.”
About Dropshot Therapeutics
Dropshot Therapeutics is an early stage biotechnology company dedicated to developing innovative RNA-based therapeutics for heart and kidney diseases. The company is based near Boston, USA and was co-founded in 2023 by Kenneth R. Chien, co-founder of Moderna and former Professor at Karolinska Institutet, and Marijn E. Dekkers, Chairman of Novalis LifeSciences and former CEO of Bayer and of Thermo Fisher Scientific.
Contact information:
etherna: media@etherna.be
Dropshot: info@dropshottherapeutics.com