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Liposome Formulation for Gene Therapeutic Research

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Liposomes are non-viral vectors composed of lipids, capable of delivering genes to cells. These versatile carriers can be customized using different lipids, targeting molecules, and other components. CD Formulation has developed a sophisticated platform to optimize liposome applications in gene therapy, offering researchers tailored solutions.

Advantages of Liposomes in Gene Therapy

Liposomes provide several benefits compared to viral vectors, including:

  • Biocompatibility: Biodegradable and low-immunogenicity, liposomes are safe for gene delivery.
  • DNA Protection: Liposomes protect DNA from enzymatic degradation and inactivation.
  • Efficient Delivery: Capable of encapsulating large amounts of DNA, liposomes improve stability and facilitate the transport of sizable DNA fragments.
  • Targeted Delivery: Liposomes can be modified for cell-specific targeting, with techniques such as PEGylation enhancing tissue penetration.

Main Applications of Liposomes in Gene Therapeutic Research

Applications Case Studies
Nucleic Acid Therapies
  • Liposome formulation for nucleic acid therapies involves the encapsulation of nucleic acids, such as DNA or RNA, within lipid-based vesicles known as liposomes. These liposomes act as delivery vehicles, protecting the fragile nucleic acids from degradation and facilitating their targeted delivery to specific cells or tissues in the body.
Case 1. Peptide-targeted liposomes containing nucleic acids
Graph illustrating the targeted evaluation process of peptide-modified liposomes.
Fig.1 Targeted evaluation of liposomes. (Agnusdei A, et al, 2024)
Therapeutic Application of CRISPR/Cas9
  • Liposomes are currently among the most extensively researched CRISPR/Cas9 delivery vectors. The surface characteristics of liposome vectors, such as surface charge, PEGylation, and ligand modification, can significantly impact gene silencing efficiency.
Case 2. Liposome in CAR-T Cell
Schematic showing the design of liposomes for CRISPR/Cas9 gene editing applications.
Fig.2 Schematic representation of liposome for Cas9 Therapy. (Supranoto YT, et al, 2020)
Immunization Therapy
  • Liposomes, modified with immune enhancers and targeting molecules, are capable of serving as a multifunctional vaccine adjuvant delivery system (VADS) capable of targeting immune cells and even organelles, inducing lysosomal escape, promoting antigen cross-presentation, and significantly enhancing vaccine efficacy while demonstrating a strong ability to induce the desired immune response.
Case 3-Vaccine adjuvant-delivery system
Diagram of a liposome-based immunomodulator for vaccine delivery.
Fig.3 Schematic representation of liposome for immunomodulator. (Wang N, et al, 2019)

Our Capabilities in Liposome-Based Gene Therapeutic Research

  • Innovative R&D : We specialize in developing novel lipid formulations, refining encapsulation methods, and optimizing targeting strategies to improve gene delivery precision and efficacy.
  • Comprehensive Characterization: We conduct thorough analyses of safety profiles, focusing on minimizing off-target effects while enhancing therapeutic outcomes.
  • Collaborative Expertise: We partner with scientific, clinical, and industry experts to translate cutting-edge research into practical applications.

Explore Our Liposome Formulation Services for Gene Therapeutic Research

Liposome Drug Delivery System Development

Our cutting-edge liposome technology holds promising applications in various drug delivery fields, such as gene therapy, vaccines, anticancer drugs, and ophthalmic formulations. Through precise control of the structure and composition of liposomes, we can achieve efficient and stable delivery of the active payload.

Liposome Drug Loading Testing

In this service, we screen suitable separation methods for the separation of free liposomes, empty loaded and drug-coated stable liposomes. We provide this service to help our customers determine the exact load and optimize the analysis of the load based on the pharmacodynamic assessment of the different load.

Custom Long-circulating Liposome Service

We can provide modification services for derivatives, and colleagues can provide PEG-modified liposomes with different chain lengths. we help our customers explore more alternative long-circulating liposomes to PEG modifications that avoid the ABC effects caused by such PEG liposomes.

Custom Polymer-modified Liposome Service

We provide screening services and provide the polymer or copolymer, polymer derivatives, including, chitosan, atelocollagen, modified xanthan gum department, alginate, cyclodextrin, gelatin, poly (acrylic acid), etc.

Liposome Formulation Development

Our formulation development services include different liposome such as injectable liposomes.

Liposome Analysis and Characterization

Liposome quality studies and CQAs guide formulation screening, stability assessments, and form the basis for further non-clinical research.

CD Formulation leads in liposome technology, offering dedicated R&D services to advance liposome-based gene delivery systems. Contact us to learn how we can support your research.

References

  1. Santos AO, da Silva LC, et al. Design of peptide-targeted liposomes containing nucleic acids. Biochimica et Biophysica Acta (BBA)-Biomembranes. 2010 Mar 1;1798(3):433-41.
  2. Supranoto YT, Nugraha MY, et al. Enhancing the potencies of chimeric antigen receptor T cell (CAR T cell) by CRISPR/CAS9 system to eradicate retinoblastoma. OISAA Journal of Indonesia Emas. 2020 Dec 20; 3(2): 73-82.
  3. Wang N, Chen M, et al. Liposomes used as a vaccine adjuvant-delivery system: From basics to clinical immunization. Journal of Controlled Release. 2019 Jun 10;303:130-50.
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