Gene Therapy Delivery Platforms
                     CD 
BProiviodep onae-srtotp ciucstlomeizsation service of 
gene  therapy for academic and industrial 
customers.
Gene therapy is developing into precise medicines that can manipulate specific genes in the 
treatment  of serious diseases and vaccines development. To effectively function in vivo, reliable 
delivery systems  are very important to the nucleic acids for protecting them from degradation and 
allowing effective  cellular uptake and release. Based on profound scientific research accumulation, 
strong R&D strength  and continuous technical support, CD Bioparticles could provide customers 
with one-stop  customization service as:
Nucleic acid services Delivery systems development Characterization
• Design • Polymer-based nanoparticles • Physical 
characterization
• Synthesis • Liposomes
• Cell characterization
• Modifications • Cell-based nanoparticles
• Animal studies
• Viral Vectors
Nucleic acid services 
platform
Design
According to the name of the disease or the gene, search the database and literature for 
the
sequence of the nucleic acid, and select the proper CDS for synthesis.
Synthesis
• In Vitro Transcription (IVT)
•Chemical synthesis for siRNA, mRNA, DNA, plasmid, ASO, sgRNA, shRNA
•The corresponding QC testing services
Modifications
5`-Terminal 3`-Terminal Code Region:
Cap0/ Cap1/Cap2, Poly(A) synthesis, m1ψ, m5C, 2'-OMe,
 Fluorescent Cap,  UTR  modification… Pseudo-Uridine, LNA, 
IRES/MS2, N6  methyladenine, 5  
UTR modification… methylcytidune…
* Other modifications if 
needed.
Delivery system development 
platforms
Polymer-based nanoparticles
Due to the wide range of sources and low immunogenicity, cationic polymers, such as PEI, 
dendrimers,
chitosan, gelatin are widely used nanoscales delivery systems for gene therapy.
Liposomes
With the similar structure of cell membrane, liposomes are considered to be a safer and 
more  effective delivery system. Its flexibility and robustness in lipid structure, composition, 
ratio and  preparation methods make liposomes an important artificial carrier for gene 
therapy. Targeting  ligands, such as peptides, antibodies have been successfully applied in 
liposomes to achieve
active targeting delivery to specified area. Furthermore, encapsulating fluorescent dyes in 
liposomes  is very beneficial for later tracking and imaging studies.
Lipid nanoparticles (LNPs) LNPs with target groups
• High nucleic acid loading • Active targeting delivery
• Stable in vivo • Tissue/cell selectivity
• Low toxicity • High bioavailability
Commercial LNPs Fluorescent liposomes
• SM-102/MC3/ALC-0315 for research only • Biological tracer
• FDA approved • Visual distinction
• Ideal delivery model
www.cd-bioparticles.net
Delivery system development 
platforms
Cell-based nanoparticles
Exosomes are nanovesicles derived from cell membrane with natural targeting ability. CD 
Bioparticles  provides various exosome production custom services for gene therapy research.
Viral vectors
Viral vectors are a tool commonly used in molecular biology to bring genetic material into cells. 
The  principle is to use the molecular mechanism of viruses to transmit their genomes into other 
cells for  infection. Its high effectiveness and tropism make viral vectors one of the most 
commonly used  carriers for gene therapy. CD Bioparticles could provide a wild range of viral 
vectors for basic  research and preclinical study.
Adenoviral vectors (Ads) Adeno-associated viral vectors (AAVs)
• Non-integrating • Non-integrating
• Large packaging capacity • Tissue tropisms
• Broad tropism • Various mutants
• Transduce (non-)dividing cells • Low immunogenicity
• High level expression • High level and long-term
Lentiviral vectors (LVs) Retroviral vectors (RVs)
• Integrating • Integrating
• Stable expression • Transduce dividing cells
• Long-term expression
www.cd-bioparticles.net
Characterization 
Platform
Physical characterization
• DNA sequencing
• Off-target detection
• Size & PDI
• Morphology
• Zeta potential
• Lipid assay (liposomes only)
• Encapsulation efficiency & Drug loading
Cell characterization
•Cell culture
•Cell targeting
•Gene & protein expression
•Titer definition
•Functional evaluation
•Pseudovirus & plasmids creation (viral vectors 
only)
•Viral vectors purification (viral vectors only)
Animal studies
•Animal model establishment
• Immunizations
•Safety evaluation
•Effectiveness evaluation
Contact 
Us
Phone: 1-631-624-4882 (USA) / 44-161-818-6441 (Europe) / Fax:1-631-938-8221
E-mail: [email protected] / www.cd-bioparticles.net
45-1 Ramsey Road, Shirley, NY 11967, USA