1. What are proprietary lipids?
Our research and development team has crafted a portfolio of ionizable lipids and compositions, leveraging years of expertise in nanoparticle formulation. These lipids allow scientists to rapidly validate drug delivery systems, accelerating clinical development and commercialization. Ionizable cationic lipids are critical to lipid nanoparticle (LNP) structure, payload delivery, and release. These lipids adjust their charge based on pH, reducing toxicity issues commonly associated with permanently cationic lipids.
We hold intellectual property rights for these lipids but offer affordable, non-exclusive clinical licenses. This includes lipid supply, LNP instruments, and related services, enabling and de-risking clinical development and commercialization.
2. How diverse is the ionizable lipid portfolio?
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Our portfolio includes over 100 ionizable lipids designed to enhance delivery efficacy and improve pharmacokinetics in RNA-LNP therapeutics. These lipids feature a range of biodegradability, with characteristic moieties that break down through natural biological processes. The ionizable cationic nitrogen head groups provide a broad pKa range, making them adaptable for different therapeutic applications. Our lipids are stable during formulation, processing, manufacturing, and storage. Several are already available for GMP activities. By collaborating with us, you can access in-house expertise for selecting the optimal ionizable lipids, customizing lipid compositions, and working to scale up GMP production. |
Fig 1: Diverse library of ionizable lipids
3. How do you select the right ionizable lipid?
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Our proprietary lipid portfolio is designed based on specific scientific applications, drug interactions, and delivery methods. Through a combination of rational design and empirical testing, we work closely with researchers to develop tailored compositions. Our lipid mixes are optimized for in vivo and ex vivo applications, maximizing performance for each specific therapeutic target. Scientists evaluate the efficacy of these formulations, selecting the lipid that ensures safe and efficient drug delivery. Collaborative, custom LNP formulation design improves the likelihood of success and maximizes the therapeutic potential of the nanoparticle. |
Fig 2: Design custom LNP formulations
4. What data supports our proprietary lipids?
Proof-of-concept data is available for various applications, including:
- Infectious disease vaccines (IM administration in mice, rats, hamsters, and non-human primates)
- Protein replacement therapy (targeted liver delivery)
- Gene editing (liver-targeted delivery)
- Ex vivo cell therapy (protein expression and gene editing)
We also offer safety and toxicology data for specific lipids from our portfolio, particularly with self-amplifying mRNA-LNP. In addition to proof-of-concept results, we have production scale-up data and demonstrated GMP manufacturing capabilities.
5. How can you get started with LNP formulation?
Our GenVoy-ILM™ platform provides pre-optimized lipid mixes designed for specific applications, streamlining screening and delivery validation. You can license these ionizable lipids through clinical stages and into commercialization. Alternatively, we offer a comprehensive evaluation process that grants access to our lipids and formulation expertise under research and collaboration agreements.
Together, we can develop custom LNP formulations and explore their potential through empirical testing. The data generated will be reviewed by our technical experts to help you select the most promising formulation for preclinical and clinical development. Our extensive in-house expertise spans from rare disease applications to large-scale clinical programs, lowering the risk and expediting the development of lipid nanoparticle formulations.
*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 Cytiva for further evaluation in clinical trials.