Future of LNPs in Developing Cell and Gene Therapies

The Future of Lipid Nanoparticles in Developing Cell and Gene Therapies

Connect with Industry Experts at the Virtual PNI Symposium

 

Join us on December 7th for the Precision NanoSystems' Symposium, a virtual event to explore how mRNA-Lipid Nanoparticles can revolutionize cell and gene therapies. This live event features scientific experts and industry leaders who will share insights into ongoing novel research and the exceptional potential of mRNA-Lipid Nanoparticles! 

 

This event is free to attend.

 

Date: December 7, 2023 

Time: 7:30 am – 9:45 am PT | 10:30 am -12:45 pm ET | 4:30 pm- 6:45 pm CET

 

The series of presentations will feature the following topics: 

 

7:30-8:15 am: mRNA-LNP Technology for Safe CAR Cell Therapy

8:15-8:45 am: mRNA-Lipid Nanoparticles for Pulmonary Delivery

8:45-9:15 am: Optimized Prime: A Transformative LNP Composition for RNA Vaccines that Scales

9:15-9:45 am: Genetic Engineering of T Cells and CD34+ HSPCs using RNA-Lipid Nanoparticle for Cell Therapies

 

Register now to save the date!

 

 

Keynote Presentation

mRNA-LNP Technology for Safe CAR Cell Therapy 

 

7:30 - 8:15 am PT | 10:30 - 11:15 am ET | 4:30 - 5:15 pm CET 


Speaker: Dr. Sandy Tretbar, Head of Cell and Molecular Biology Unit, Fraunhofer Institute for Cell Therapy and Immunology

 

Dr. Sandy Tretbar

This presentation will discuss why adoptive immunotherapy is one of the biggest innovations in modern cancer therapy using genetically modified immune cells redirected to efficiently target and eliminate cancer cells. The direct transfer of in vitro transcribed CAR-mRNA into T cells is pursued as a promising strategy for CAR T cell engineering. In current clinical trials, mRNA electroporation (EP) is used as an mRNA delivery method for the generation of CAR T cells but achieves only poor anti-tumor responses. Therefore, mRNA was optimized, and lipid nanoparticles (LNPs) were examined for ex vivo CAR-mRNA delivery which resulted in a significantly prolonged CAR T efficacy in vitro. CAR expression and in vitro functionality of mRNA-LNP-derived CAR T cells were comparable to stably transduced CAR underlining the great potential of mRNA-LNP delivery for ex vivo CAR T cell modification as the next-generation transient approach for clinical studies. For that, optimization was performed for the current lab-scale mRNA-CAR T generation process for optimal cell viability, expansion, modification, and functionality for upscaling and process development of mRNA-based CAR-T cell therapies.

 

Presentation
mRNA-Lipid Nanoparticles for Pulmonary Delivery 

8:15 - 8:45 am PT | 11:15 - 11:45 am ET | 5:15 - 5:45 pm CET 


Speaker: Dr. Sams Sadat, Scientist, Precision NanoSystems 

 

Sams Sadat

Despite the recent successes of lipid nanoparticle (LNPs)-mediated mRNA vaccines against SARS-CoV-2, inhalation-based gene therapy and mucosal immunization remain challenging with respect to treating various lung diseases (e.g., inherited diseases, pulmonary hypertension, asthma, cystic fibrosis, cancers, etc.). Systemic therapeutic applications of currently available lipid and non-lipidic nanoparticulate platforms have been investigated mostly for liver diseases because of their favorable accumulation tendency into the liver, which in due course restricts their access to other organs, including the lungs. The intranasal (IN) administration, aerosol inhalation, and intratracheal instillation of nucleic acids-encapsulated nanoparticles can directly access the pulmonary system, principally lung epithelium. 

 

In this respect, we demonstrate the preclinical success of the direct IN delivery of mRNA-encapsulated LNPs that were pre-mixed with our newly invented lung-targeting IN solution. 

 

Presentation 
Optimized Prime: A Transformative LNP Composition for RNA Vaccines that Scales

8:45 - 9:15 am PT | 11:45 - 12:15 pm ET | 5:45 - 6:15 pm CET

Speaker: Dr. Natalie Orr, Scientist, Product Development, Precision NanoSystems

 

Natalie Orr

Lipid nanoparticle encapsulated RNA (RNA-LNP) vaccines have played a pivotal role in the global response to the COVID-19 pandemic. With proven safety and efficacy, RNA-LNPs are now at various stages of development against a variety of other infectious diseases. However, limited access to potent ionizable lipids, a key component in LNPs, and the expertise required to formulate and scale LNPs present real barriers to entry in this field. To overcome these challenges, we developed and optimized a novel ionizable lipid and a corresponding LNP composition for the delivery of RNA in vaccine applications. Using a scalable mixing platform, we formulated vaccine candidate LNPs with both mRNA and self-amplifying RNA and investigated their physicochemical attributes and in vivo immunogenicity.

 

 

Presentation 
Genetic Engineering of T Cells and CD34+ HSPCs using RNA-Lipid Nanoparticle for Cell Therapies

9:15 - 9:45 am PT I 12:15 - 12:45 pm ET I 6:15 - 6:45 pm CET 

Speaker: Dr. Angela Zhang, Product Management Leader, Precision NanoSystems

 

Angela Zhang

Delivering nucleic acids using lipid nanoparticles (LNP) to target cells has tremendous potential to enable new gene and cell therapies. However, limited access to novel LNP solutions beyond vaccines can be a barrier to adoption. Herein, we describe a library of ionizable lipids that can be used as functional excipients for nucleic acid delivery for different routes of administrations. To exemplify the benefits of LNPs in the production of cell-based therapies, we demonstrate a robust method for the multi-step engineering of gene-edited CAR T cells and CD34+ hematopoietic stem and progenitor cells (HSPCs) using off-the-shelf LNP reagents with a scalable path to the clinic.