Lipid & Gene Therapy Solutions

In recent years, lipids have attracted attention for their potential use in gene delivery. Gene delivery is the process of introducing foreign genetic material into cells to treat genetic disorders, cancer, and other diseases. Lipids offer several advantages for gene delivery, including their biocompatibility, the ability to form stable complexes with genetic material, and the potential for targeted delivery to specific tissues. Leveraging BOC Sciences' strong supply capabilities in the field of lipids and PEG, we have launched a one-stop lipid solution for gene therapy and delivery, including lipid design and synthesis, lipid characterization analysis, lipid formulation and lipid nanoparticles development support.

Fig. 1. Lipid nanoparticles for drug and gene delivery (Bioconjugate Chemistry. 2022, 1043-1802).

Liposomes in Gene Therapy

In gene therapy, genetic material, such as plasmid DNA, antisense oligonucleotides, and small interfering RNA (siRNA), can be used to treat or prevent disease. This includes replacing a mutated gene, inactivating a mutated gene, or introducing a new gene. Although gene therapy is a promising treatment option for many diseases, including genetic disorders, certain types of cancer, and certain viral infections, successful gene therapy has been hampered by the lack of effective delivery systems (viral and non-viral ) and be hindered. Several non-viral gene vectors, such as lipids, polymers, and peptides, can be used for this purpose. Liposome delivery of nucleic acids (e.g., plasmid DNA, asODN, and siRNA) represents a very promising nanocarrier system for relatively safe and efficient gene delivery to target locations in the body. Lipid-based delivery systems have also been shown to be stable in serum and plasma, improve biodistribution, extend circulation half-life, and enhance target tissue selectivity. The most common lipids in liposomes are cationic lipids, which promote binding between positively charged head groups and negatively charged nucleic acids.

Lipid Nanoparticles for Gene Delivery

The use of lipid nanoparticles (LNPs) in COVID-19 mRNA vaccines has renewed attention as a gene delivery vehicle. LNPs used for gene therapy are usually nanoparticles composed of ionized cationic lipids, phospholipids, cholesterol and PEG lipids. Because liposomes had long been approved by regulatory agencies for clinical use in small molecule drugs, they were quickly identified as a potential delivery vehicle by gene therapy pioneers. BOC Sciences offers a variety of lipid nanoparticles designed specifically for gene delivery applications. Our lipid nanoparticles are available in different formulations and sizes, allowing researchers to choose the best option for their specific gene delivery needs. Our lipid nanoparticles support the delivery of a variety of nucleic acids, including antisense oligonucleotides, plasmid DNA, messenger RNA, small interfering RNA, and microRNA.

Our Lipid Solutions for Gene Therapy

Design and Custom Synthesis of Lipids

One of the key services provided by BOC Sciences is the design and custom synthesis of lipid-based gene delivery systems. Our team of experts works closely with customers to develop custom lipid nanoparticles, liposomes, or other lipid-based vehicles that target specific genetic material (antisense oligonucleotides, plasmid DNA, messenger RNA, small interfering RNA, and microRNA ) delivery has been optimized. By leveraging our expertise in lipid chemistry and drug delivery, BOC Sciences can help customers overcome the challenges associated with gene delivery and improve the effectiveness and safety of their therapeutic interventions.

Analysis and Characterization of Lipids

BOC Sciences also provides characterization and evaluation services for a range of lipids to evaluate the performance of lipid-based gene delivery systems. In addition to conventional characterization of lipid composition stability, particle size distribution, loading efficiency, etc., we also support preclinical in vitro and in vivo studies to evaluate the stability, transfection efficiency and cytotoxicity of lipid nanoparticles, as well as their targeting capabilities of a specific cell type or tissue.

Lipid Formulation and Optimization

In addition, BOC Sciences provides formulation and optimization services to help customers fine-tune their lipid-based gene delivery systems for optimal performance. This includes developing scalable and repeatable manufacturing processes, as well as optimizing formulation parameters to achieve desired treatment outcomes. By leveraging its expertise in formulation development and process optimization, BOC Sciences can help customers streamline the development of gene delivery systems and accelerate their translation into clinical applications.

Our PEG Solutions for Gene Delivery

PEG is a highly flexible and hydrophilic compound. Its anti-phagocytic effect on macrophages, resistance to immune recognition, non-binding to proteins, and good biocompatibility make PEG-terminated liposomes widely used in drug delivery. PEG-terminated liposomes have attracted widespread attention as passive targeted drug delivery carriers for the treatment of cancer and infectious diseases. They are superior to other carriers in increasing the systemic circulation time of drugs, delivering active molecules to the site of action, and protecting healthy tissue from damage by toxic effects. During the preparation process, a key step in developing long-circulating liposomes is the addition of synthetic polymer PEG, such as PEG-DSPE, to the liposome composition. Based on this, our strong research capabilities in the field of PEG also support one-stop solutions for gene delivery. Our services include:

• Custom development and synthesis of functionalized and structured PEGs
• PEG hydrogel development services
• PEGylation of lipid nanocarriers
• PEGylation of nucleic acids (DNA, mRNA, siRNA, oligonucleotides, etc.)
• Scheme screening for PEGylation of gene vectors
• Characteristic evaluation and safety evaluation
• Assay development and screening

BOC Sciences' lipid gene delivery services are supported by an unwavering commitment to quality and compliance with regulatory standards. Our state-of-the-art facilities are equipped with the latest technology and adhere to strict quality control measures to ensure the safety and effectiveness of our products and services. Additionally, our team of experts is well-versed in the regulatory requirements for gene therapies and can provide professional guidance and support as clients navigate the complexities of drug development and commercialization.

References

1. Poellmann, M.J. et al. Dendritic-Linear Copolymer and Dendron Lipid Nanoparticles for Drug and Gene Delivery. Bioconjugate Chemistry. 2022, 1043-1802.
2. Nordling-David, M.M et al. Gene Delivery by Liposomes. Isr. J. Chem. 2013, 53(9-10): 737-747.