Gene Therapy Drug Delivery Systems for Biologics

Gene therapy drug delivery systems enable the controlled distribution of gene expression systems in vivo, targeting specific cells and ensuring intracellular trafficking and nuclear translocation. These systems protect gene therapies from premature degradation and facilitate cell-specific delivery to target organs. At CD Formulation, we specialize in custom gene therapy drug delivery systems based on lipids, polymers, and lipid-polymer hybrid nanoparticles.

Gene Therapy Drugs and Vector Advantages

Gene therapy drugs mainly include plasmid DNA, small interfering RNA (siRNA), microRNA (miRNA), short hairpin RNA (shRNA), antisense oligonucleotides (ASO), and CRISPR/Cas9 systems. Gene therapy drug carriers have the ability to protect nucleic acid drugs, extend half-life, improve targeting, and enhance endosomal escape and intracellular release.

Fig.1 Gene therapy drugs. (Xiuhua Pan, et al. 2021)

With the rapid development of materials science and nanotechnology, many new viral and non-viral vectors have been used for gene therapy drug delivery.

• Viral vectors mainly include adeno-associated virus (AAV), adenovirus (AdV), lentivirus (LV) and bacteriophage. Viral vectors for gene therapy drugs have the advantages of high transfection efficiency and stable transgene expression.
• Non-viral vectors mainly include cationic liposomes, micelles, polymers and inorganic nanoparticles. With the rise of biomaterials, innovative nanocarriers include DNA nanocores, metal organic skeletons, cell membranes, etc. With the deepening of research, non-viral vectors have the advantages of large gene load, low immunogenicity, and high safety in large-scale production. Moreover, modified nanocarriers can not only increase drug loading and gene therapy effects, but also induce higher biosafety and higher targeting characteristics.

Applications for Gene Therapy Drug Delivery Systems

Gene therapy has the potential to treat genetic disorders, cancer, viral infections, and cardiovascular diseases by modifying disease-causing genes.

• Cancer Therapy: RNA interference (RNAi) offers potent gene silencing for cancer treatment.
• Duchenne Muscular Dystrophy: CRISPR/Cas9 restores gene coding frames, enabling mutation correction in Duchenne Muscular Dystrophy.
• Parkinson's Disease: CRISPR-Cas9 technology targets specific brain regions to alter function and treat neurological disorders.

Our Gene Therapy Drug Delivery Technologies for Biologics

CD Formulation is committed to exploring and researching gene therapy drug delivery systems for biologics, including lipid delivery systems for gene therapy, polymeric delivery systems for gene therapy, and lipid-polymer hybrid delivery systems for gene therapy.

Lipid Delivery System for Gene Therapy

Changing the composition of the lipids in lipid nanoparticles can lead to more specific delivery of lipid nanoparticles to certain organs, such as the brain. Lipid nanocarriers are potentially good colloidal drug carriers for gene delivery. CD Formulation has been committed to developing lipid delivery systems for gene therapy, including lipid nanoparticle delivery systems...

Polymeric Delivery System for Gene Therapy

Polymers are ideal biomaterial platforms due to their scalability, versatility, and exquisite tunability to efficiently deliver nucleic acid payloads while minimizing immune responses and cytotoxicity. High-performance polymer carriers for gene therapy can achieve therapeutic goals for both in vivo and in vitro gene therapy. CD Formulation has experience in polymeric delivery systems R&D in gene therapy...

Lipid-Polymer Hybrid Delivery System for Gene Therapy

Lipid-polymer hybrid nanoparticles have enhanced stability, prevention of drug leakage, the possibility of easy surface functionalization, and targeting ability, which can improve their physicochemical properties and cell binding, such as cell membrane permeability and immune response, for delivery vehicles of gene therapy. CD Formulation has lots of experience in constructing lipid-polymer hybrid delivery systems in gene therapy...

Our Development Approaches

Gene therapy is one of the promising therapies that can treat many diseases such as genetic diseases, viral infections and cancer using regulatable genes. Therefore, it is crucial to construct gene therapy drug delivery systems for gene therapy of human genetic diseases and other diseases. For gene therapy drug delivery systems, our research goal is to develop vectors such as viral and non-viral vectors.

Viral Vector Gene Delivery Systems

The viruses we use for gene therapy drug delivery systems include retroviruses, adenoviruses, adeno-associated viruses, and herpes simplex viruses. Viral gene delivery systems mainly include viral vectors based on DNA, RNA and oncolytic virus vectors.

Fig.2 Viral vector gene delivery systems. (CD Formulation)

• In Vivo Delivery: Genetic material is directly delivered to target tissues.
• In Vitro Delivery: Genes are introduced into bone marrow or other target cells.
• In-Situ Delivery: Genetic material is administered into the target tissue.

Non-viral Vector Gene Delivery Systems

Non-viral gene delivery systems include physicochemical methods such as physical methods and chemical methods.

• Non-viral physical gene delivery systems are artificially mediated by physical methods to introduce genetic material through the cell membrane. Our more mature physical methods of gene delivery include needle injection, ballistic DNA injection, sonoporation, photoporation, magnetic infection and hydroporation.
• Non-viral chemical gene delivery systems use synthetic or natural compounds to form particles (such as liposomes and polymers) to facilitate gene transfer into cells.

Why Choose CD Formulation?

• Tailored Solutions: Our expertise in lipids, polymers, and hybrid nanoparticles allows us to develop customized non-viral vectors to meet client-specific needs.
• Advanced Technologies: We apply cutting-edge nanotechnology to create safe, effective delivery systems for gene therapies.
• Viral and Non-Viral Expertise: We excel at constructing viral and non-viral vectors tailored for gene therapy drug delivery.

Published Data

With the development of molecular biology, molecular genetics, clinical medicine and emerging nanomaterials, gene therapy drug delivery systems have been continuously optimized and matured and have been widely used in clinical practice. CD Formulation as a world-class nanoformulation servicing company, can develop gene therapy drug delivery systems for biologics based on lipids, polymers and lipid-polymer hybrid nanoparticles. If you are interested in our gene therapy drug delivery systems for biologics, please do not hesitate to contact us.

How It Works

STEP 1

Submit a service request describing your specific research or development need.

STEP 2

We'll email you to provide your quote and confirm order details if applicable.

STEP 3

Execute the project with real-time communication, and deliver the final report promptly.