Gene Therapy Delivery Platforms

Excipients for mRNA Delivery Download PDF

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

Design
Synthesis
Modiﬁcations

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

Modiﬁcations

5`-Terminal
Cap0/ Cap1/Cap2,Fluorescent Cap,IRES/MS2,UTR modification…

3`-Terminal
Poly(A) synthesis, UTRmodification…

Code Region:
m1ψ, m5C, 2'-OMe,Pseudo-Uridine, LNA, N6methyladenine, 5methylcytidune…

* 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.

Gene Therapy Delivery Platforms

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

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.

Gene Therapy Delivery Platforms

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)

S Integrating
Stable expression
Long-term expression

Retroviral vectors (RVs)

Integrating
Transduce dividing cells

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

Brochures

USA

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