Nanoparticle Delivery System Development

Nanoparticles (NPs) are colloidal drug delivery systems that include particles with diameters ranging from 10 to 1000 nanometers. The combination of gene therapy and nanodelivery systems has broadened the therapeutic and biomedical applications of these molecules, such as bioanalysis, gene silencing, protein replacement, and vaccines. In recent years, nanoparticles have been used in a variety of medical and biological research applications, including the field of gene therapy. CD Formulation can provide clients with nanoparticle delivery system development to protect genetic material, improve gene delivery efficiency, and advance gene therapy research.

Advantages of Nanoparticle Delivery System

The key advantages of nanoparticles are that they increase bioavailability through enhanced water solubility, and they increase resistance time in vivo, as well as targeting the drug to specific sites of action in the body. This leads to a corresponding reduction in the dose and toxicity of the drug required, allowing for the safe delivery of therapeutic drugs and protection of non-target tissues and cells from serious side effects.

• Reproducibility and safety. Synthetic nanoparticle technology offers a better safety profile than viral vectors, although a small portion of the population may develop immune, inflammatory, or allergic reactions to certain components of the formulation. However, for the majority of the population, synthetic particles exhibit better immune privileges than viral vectors, allowing for a limited number of repeat administrations.
• High capacity. Synthetic nanoparticles can encapsulate most current load types, and as load sizes continue to increase, nanodelivery vectors with higher capacities can be custom synthesized.
• Targeting. Synthetic particles can offer strong potential for gene therapy for liver diseases. Additional cell tropism and biodistribution properties can be engineered to some extent through chemical or electrostatic modifications or incorporation of cell surface proteins.

Our Services for Developing Nanoparticle Delivery System

Design phase

In this phase, we need to identify the goals and requirements of the delivery system and select suitable nanocarrier materials such as liposomes, polymers, peptides, solid lipid nanoparticles (SLNs), and polymeric micelles.

Synthesis and functionalization

Synthesis of nanoparticles and their functionalization to enhance their stability, targeting and cellular uptake. This may include surface modification to increase circulation time, or attachment of targeted ligands to improve delivery efficiency to specific tissues or cell types.

In vitro testing

We performed preliminary in vitro tests in cell culture to evaluate the transfection efficiency, cytotoxicity and specificity of the nanodelivery system. These tests help to screen and optimize promising nanoparticle formulations.

In vivo model evaluation

Subsequently, we evaluate the pharmacokinetics, tissue distribution, targeting efficiency and safety of the nanodelivery systems in animal models. This often involves high-throughput screening to rapidly identify effective delivery systems.

Preclinical optimization

This process is based on the results of the in vivo model, and we further optimize the nanodelivery system to improve its efficacy and safety and reduce potential side effects.

Optimizing Nanoparticle System for Gene Therapy and Editing

• We can optimize the stability, encapsulation efficiency, and cellular uptake of nanodelivery systems by engineering them to contain a variety of lipids, including cationic lipids, neutral lipids, and cholesterol. In addition, nanoparticles can be functionalized with targeted ligands to improve specificity for certain cell types or tissues. Studies have demonstrated the use of nanoparticle-delivered gene therapy to treat a variety of diseases. For example, myotonic dystrophy, dystrophies, etc. can be treated using nanodelivery systems.
• In addition to gene therapy, nanoparticles have been used to deliver gene editing tools such as CRISPR-Cas9 and base editors. Lipid nanoparticles have been tested for delivery of base editors or CRISPR-Cas9 systems that delete the BCL11A gene to treat β-thalassemia and sickle cell disease in human subjects.

Our Platforms for Nanoparticle Delivery System Development

Highlights of Our Nanoparticles Delivery System Development Service

• With our rich expertise and years of development experience, we can provide our customers with universal research including production and characterization of nanostructured multiple carriers.
• We develop nanodelivery system platforms and can formulate personalized nanodelivery carrier strategies according to the different needs of our customers.
• We have a first-class research and development team to rapidly and powerfully advance the research and application of nanodelivery systems in gene therapy.
• Our state-of-the-art laboratory equipment can support researchers to effectively advance the development of nanodelivery systems in gene therapy, including but not limited to a wide range of nanocharacterization instruments.

Published Data

CD Formulation is dedicated to the development of gene therapy formulations. Based on our state-of-the-art platform and specialized experts, we can provide reliable and professional nanodelivery system development services in R & D, manufacturing and quality control for nanodelivery systems in gene therapy. If you are interested in us, please feel free to contact us.