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Testing the In Vitro and In Vivo Efficiency of mRNA-Lipid Nanoparticles Formulated by Microfluidic Mixing
Published on: January 20, 2023
11.8K
Lipid-based nucleic acid therapeutics with in vivo efficacy
1Department of Pharmaceutical Sciences and Moulder Center for Drug Discovery Research, School of Pharmacy, Temple University, Philadelphia, Pennsylvania, USA.
Wiley Interdisciplinary Reviews. Nanomedicine and Nanobiotechnology
|September 30, 2022
Summary
Lipid-based systems are advanced synthetic vectors for delivering nucleic acids, offering advantages over viral methods. This review covers their development, formulation, manufacturing, and clinical applications in medicine.
Area of Science:
- Nanomedicine
- Drug Delivery
- Biotechnology
Background:
- Synthetic vectors offer advantages over viral counterparts for nucleic acid delivery, including reduced immunogenicity and large payload capacity.
- Lipid-based systems have demonstrated significant clinical success, notably with siRNA therapeutics and mRNA vaccines.
- These systems are maturing into a versatile technology for delivering diverse nucleic acid payloads like DNA, RNA, and oligonucleotides.
Purpose of the Study:
- To provide a comprehensive review of lipid-based nucleic acid delivery systems.
- To discuss the mechanisms, delivery barriers, and key factors influencing therapeutic success.
- To highlight formulation, manufacturing, preclinical, and clinical aspects of these advanced nanocarriers.
Main Methods:
- Literature review of lipid-based nucleic acid delivery systems from 1993 to present.
- Discussion of payload properties, lipid components, and formulation parameters.
- Analysis of manufacturing techniques, preclinical applications, and clinical outcomes.
Main Results:
- Lipid-based systems are effective for delivering various nucleic acid payloads (pDNA, ASO, siRNA, miRNA, saRNA, mRNA).
- Key factors influencing efficacy and safety include payload structure, lipid composition, and formulation parameters.
- Manufacturing advancements enable reproducible and scalable production of these nanoparticles.
Conclusions:
- Lipid-based nucleic acid delivery represents a mature and impactful technology in therapeutics.
- Understanding physicochemical properties, manufacturing, and clinical performance is crucial for successful translation.
- These systems hold significant promise for future therapeutic approaches and drug discovery.
