Top 5 Things to Know about the Lipid Nanoparticle Portfolio
What are Precision NanoSystems’ Proprietary Lipids?
Precision NanoSystems enables the world’s leading drug developers to create genomic medicines by offering NanoAssemblr® instruments, reagents, and BioPharma services.
Our R&D team has designed and developed a portfolio of ionizable lipids and compositions that integrate years of trusted nanoparticle expertise into optimized formulations enabling any scientist to easily validate drug delivery, accelerating clinical development and commercialization. Ionizable cationic lipids play an integral role in assembly of the lipid nanoparticles (LNPs), delivery and release of the payload, leveraging its pH sensitivity to maintain neutral charge in physiological conditions, eliminating the main source of toxicity associated with permanently cationic lipids.
While Precision NanoSystems owns the intellectual property pertaining to these lipids, we offer affordable, non-exclusive clinical licenses combined with lipid supply, LNP instrumentation and services to enable, accelerate and de-risk clinical development and commercialization.
How Diverse is the Ionizable Lipid Portfolio?
Precision NanoSystems offers over a hundred ionizable lipids developed to optimize delivery efficacy and pharmacokinetic profiles of RNA-LNPs therapeutics. Varied structural features of the ionizable lipids offer a spectrum of biodegradability, using characteristic moieties that degrade in biological environments through hydrolytic metabolic processes. While an ionizable cationic nitrogen head group provides an extensive pKa range, making them functionally active for different applications.
The ionizable lipids are stable under typical LNP formulation, processing, manufacturing and storage conditions, with certain lipids are already available for GMP activities. Establishing a partnership with Precision NanoSystems leverages our in-house expertise for ionizable lipid selection, customizing fit-for-purpose lipid compositions, and our strong working relationships with GMP manufacturers, to produce GMP lipids at scale for commercial processes.
What is the Process to Select an Appropriate Ionizable Lipid?
The proprietary lipid portfolio is well-characterized based on scientific applications, drug substance interactions, and delivery routes. Through a combination of rational design and empirical experimentation, the Precision NanoSystems team works collaboratively with scientists to develop fit-for-purpose compositions. Based on years of formulation experience, we supply lipid mixes catered to the intended in vivo/ex vivo application, designed to maximize performance.
Scientists will then evaluate the performance and delivery efficiency, selecting the ionizable lipid formulation that delivers a safe and effect drug substance. Partnering together to rationally design and test custom LNP formulations, improves the chances of success and reaching the full potential of targeted nanomedicines
What Data is Available for Precision NanoSystems Proprietary Lipids?
Proof-of-concept data is available for the following applications:
Infectious Disease Vaccine (IM administration, across multiple species: mice, rats, hamsters and NHPs)
Protein replacement therapy (Liver delivery)
Gene editing (Liver delivery)
Ex-vivo cell therapy (Protein expression and gene editing)
In 2020, Precision NanoSystems was funded by the Canadian Government to establish COVID vaccine candidates through the Canadian Strategic Innovation Fund (SIF). Under this program, we have generated safety and toxicology data for certain ionizable lipids from our portfolio using self-amplifying mRNA-LNPs. Beyond proof-of-concept and validation data, we also have scale-up production data and a proven capability to manufacture GMP batches of the vaccine.
How Do I Get Started?
The GenVoy-ILM™ platform offers screening and pre-optimized lipid mixes for specific applications to leverage our internal expertise for lipid selection. These LNP formulations accelerate screening and delivery validation, using ionizable lipids that can be licensed through clinical stages to commercialization. Alternatively, an evaluation process offers access to the ionizable lipids and formulation team with research and collaboration terms.
Together, custom LNP formations are developed and empirically explored. Resultant data may be shared and examined with our technical experts to help make an informed decision on the most promising formulation to proceed for late preclinical and clinical development. Our team has expertise ranging from collaborations for small-scale rare disease applications with unmet therapeutic needs to robust clinical applications with global implications. This deep in-house expertise helps lower the risk of developing bespoke lipid nanoparticle formulations across applications.
All off-the-shelf ionizable lipid formulations are intended for research use only and not for in-human use. After appropriate efficacy and safety evaluations in preclinical settings, ionizable lipids and related compositions can be licensed from Precision NanoSystems for further evaluation in clinical trials.