Lipid Nanoparticles (LNPs) in Gene Therapy
2BScientific are proud to introduce Lipid Nanoparticles (LNPs) from Progen as a new addition to our cell and gene therapy product portfolio. LNPs are a non-viral delivery system that overcomes some of the key limitations associated with viral-based gene delivery methods. Viral vectors, such as Adeno-Associated Virus (AAV), have been widely used for gene therapy, and in some cases, are effective for their suited application however, they come with challenges including immune responses and limited scalability. LNPs provide a safer and more flexible approach, facilitating efficient delivery of genetic material while protecting fragile nucleic acids (Takanashi et al., 2023). Unlike viral vectors, LNPs do not integrate into the host genome, reducing the risk of insertional mutagenesis and off-target effects.
How do LNPs Compare to AAVs?
In past studies, AAV-based gene therapy has demonstrated significant clinical success, yet its potential for triggering immune responses remains a concern. This can limit repeated dosing and require immunosuppressive strategies, which may introduce additional risks. In contrast, LNPs are non-immunogenic, meaning they are less likely to elicit an immune response, making them a preferred choice for repeated administrations (Ronzitti, Gross & Mingozzi, 2020). Additionally, LNPs are easier to scale up for production, making them a more cost-effective and viable option for clinical applications.
Feature | Viral (AAV) | Non-Viral (LNPs) |
Advantages | High transfection efficiency, long-lasting expression, targeted delivery to specific tissues | Broad payload flexibility (mRNA, DNA, CRISPR), reduced immune response, easier scalability |
Challenges | Limited genetic payload, potential immune reaction, complex manufacturing | Lower transfection efficiency in some cell types, formulation stability challenges |
Applications of LNPs
LNPs are recognised for their role in RNA-based therapeutics, notably in mRNA vaccines. Yet they do have a broader range of applications including:
- Delivery of small molecules, RNA, proteins, and peptides: LNPs serve as efficient carriers for a variety of therapeutic agents, ensuring their stability and targeted release in the body.
- Gene editing tools (e.g. CRISPR-Cas9 systems): The ability of LNPs to encapsulate and deliver gene-editing components directly to cells enhances the precision and efficacy of genome modification.
- Targeted drug delivery for various diseases: LNPs can be engineered for tissue-specific delivery, increasing the effectiveness of treatments while minimising systemic side effects.
By reducing the immune response, LNPs are particularly valuable for in vivo applications, ensuring safer and more effective gene therapies (Pardi & Ivics, 2024). Their biodegradable nature further enhances their safety, reducing the risk of long-term build up in the target. Whether you are working on vaccine development, rare genetic disorders, or next-generation therapeutic solutions, LNPs offer a highly promising and adaptable platform.
PROGEN’s LNP Delivery Kits and LNP Protein Delivery Kits | ||
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References
Takanashi, A., Pouton, C.W. and Al-Wassiti, H., 2023. Delivery and expression of mRNA in the secondary lymphoid organs drive immune responses to lipid nanoparticle-mRNA vaccines after intramuscular injection. Molecular Pharmaceutics, 20(8), pp.3876-3885.
https://doi.org/10.1021/acs.molpharmaceut.2c01024
Ronzitti, G., Gross, D.A. and Mingozzi, F., 2020. Human immune responses to adeno-associated virus (AAV) vectors. Frontiers in Immunology, 11, p.670.
https://doi.org/10.3389/fimmu.2020.00670
Pardi, N. and Ivics, Z., 2024. An LNP-CRISPR gene editing drug demonstrates efficacy and safety in patients with hereditary angioedema following in vivo administration. Journal of Allergy and Clinical Immunology, 154(2), pp.272-274.
