Nanoparticle Vector Development Platforms
Nanoparticles' ability to form stable interactions with ligands, their variability in size and shape, high payload capacity, and the convenience of binding hydrophilic and hydrophobic substances make them an advantageous platform for the targeted and controlled delivery of small molecules and large molecules in rare disease treatment. As a company with extensive experience in the field of gene delivery, our company is dedicated to providing nanoparticle delivery system development services for rare disease therapy research and development.
Delivery Ability of Nanoparticles
Nanoparticles are tiny particles with diameters in the nanoscale range. The characteristics of nanoparticles make them a potentially powerful tool for creating new materials, drug delivery systems, sensors, and many other applications. Their properties and applications vary based on their size, shape, surface properties, and the materials used.
The advantage of co-delivery of nanoparticles with multiple materials (Nano-structure materials) is the ability to combine the properties and functions of different materials for more efficient, precise, and versatile drug delivery and rare disease therapy. Nano-structure materials can be divided into three categories:
- Polymeric nanoparticles
- Non-polymeric nanoparticles
- Lipid nanoparticles
Fig.1 Schematic representations of the therapeutic nanoparticles. (Yetisgin, Abuzer Alp, et al., 2020)
Nanoparticle Vectors for Rare Diseases
Gene therapy has emerged as one of the most promising biomedical technologies for rare diseases. However, target cells cannot effectively internal naked genetic molecules due to their sensitivity to serum nucleases, rapid renal clearance, uptake by phagocytic cells, reduced uptake by target cells, and toxicity. Nanomaterial vectors for gene delivery have garnered worldwide attention. Some nanoparticle vectors used for gene delivery have also initiated early-stage research of rare diseases.
Table 1. List of nanoparticle vector delivery systems for rare diseases.
(Yetisgin, Abuzer Alp, et al., 2020)
| Name of siRNA Therapeutics or siRNA | Delivery Material | Administration Route | Indication/Disease model | Target Organ | Research Progress |
|---|---|---|---|---|---|
| Patisiran (ALNTTR02) | LNP (lipid and lipid-derived nanoparticle) | i.v. | Hereditary transthyretin-mediated amyloidosis (hATTR) | liver | FDA approved (ONPATTRO™) |
| Tmprss6 siRNA | Ionizable LNP | i.v. | β-Thalassemia | liver | Pre-clinical |
Our Services
With advanced technology and a professional team, our company provides nanoparticle vector development and vector evaluation services, providing you with safe and more effective therapeutic nanoparticle vectors to accelerate your research and development of rare disease therapies.
The services we provide include but are not limited to:
Based on different diseases, our company develops nanoparticle carriers with suitable particle sizes and shapes to make them easier to deliver and extend their aggregation time. In addition, we provide services for the modification of nanoparticle surfaces with polyethylene glycol (PEG), carbohydrates, acetyl groups, or protein moieties (arginine-glycine-aspartate (RGD) peptides, albumin) to alter retention times and improve bioavailability.
Project Workflow
With leading technology and many years of successful experience in the development of gene delivery tools, our company is well-positioned to provide customers with nanoparticle vector development services for the gene therapy of rare diseases. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
Reference
- Yetisgin, Abuzer Alp, et al. "Therapeutic nanoparticles and their targeted delivery applications." Molecules 25.9 (2020): 2193.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.