Gene Therapy Delivery Platforms

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CD Bioparticles
Provide one-stop customization service of gene
therapy for academic and industrial customers.
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Gene therapy is developing into precise medicines
that can manipulate speci?c 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 scienti?c
research accumulation, strong RD strength and
continuous technical support, CD Bioparticles
could provide customers with one-stop
customization service as

• Nucleic acid services
• Design
• Synthesis
• Modi?cations

• Delivery systems development
• Polymer-based nanoparticles
• Liposomes
• Cell-based nanoparticles
• Viral Vectors

• Characterization
• Physical characterization
• Cell characterization
• Animal studies

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
• Modi?cations

Code Region m1?, m5C, 2'-OMe, Pseudo-Uridine,
LNA, N6 methyladenine, 5 methylcytidune
5-Terminal Cap0/ Cap1/Cap2, Fluorescent Cap,
IRES/MS2, UTR modi?cation
3-Terminal Poly(A) synthesis, UTR modi?cation
Other modi?cations if needed.
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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 ?exibility and robustness in
lipid structure, composition, ratio and
preparation methods make liposomes an important
arti?cial carrier for gene therapy. Targeting
ligands, such as peptides, antibodies have been
successfully applied in liposomes to
achieve active targeting delivery to speci?ed
area. Furthermore, encapsulating ?uorescent dyes
in liposomes is very bene?cial for later
tracking and imaging studies.

• Lipid nanoparticles (LNPs)
• High nucleic acid loading
• Stable in vivo
• Low toxicity

• LNPs with target groups
• Active targeting delivery
• Tissue/cell selectivity
• High bioavailability

• Commercial LNPs
• SM-102/MC3/ALC-0315 for research only
• FDA approved
• Ideal delivery model

• Fluorescent liposomes
• Biological tracer
• Visual distinction

www.cd-bioparticles.net
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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)
• Non-integrating
• Large packaging capacity
• Broad tropism
• Transduce (non-)dividing cells
• High level expression

• Adeno-associated viral vectors (AAVs)
• Non-integrating
• Tissue tropisms
• Various mutants
• Low immunogenicity
• High level and long-term

• Lentiviral vectors (LVs)
• Integrating
• Stable expression
• Long-term expression

• Retroviral vectors (RVs)
• Integrating
• Transduce dividing cells

www.cd-bioparticles.net
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Characterization Platform

• Physical characterization
• DNA sequencing
• Off-target detection
• Size PDI
• Morphology
• Zeta potential
• Lipid assay (liposomes only)
• Encapsulation ef?ciency Drug loading
• Cell characterization
• Cell culture
• Cell targeting
• Gene protein expression
• Titer de?nition
• Functional evaluation
• Pseudovirus plasmids creation (viral vectors
  only)
• Viral vectors puri?cation (viral vectors only)
• Animal studies

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Contact Us
Phone [phone] (USA) / 44-161-818-6441
(Europe) / Fax[phone] E-mail
info_at_cd-bioparticles.com / www.cd-bioparticles.net
45-1 Ramsey Road, Shirley, NY 11967, USA