Gene Therapy Delivery Platforms


Presentation Transcript

  • 1.CD Bioparticles Provide one-stop customization service of gene therapy for academic and industrial customers.
  • 2.Nucleic acid services platform 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 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 * Other modifications if needed. 5`-Terminal Cap0/ Cap1/Cap2, Fluorescent Cap, IRES/MS2, UTR modification… 3`-Terminal Poly(A) synthesis, UTR modification… Code Region: m1ψ, m5C, 2'-OMe, Pseudo-Uridine, LNA, N6 methyladenine, 5 methylcytidune…
  • 3.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) High nucleic acid loading Stable in vivo Low toxicity Commercial LNPs SM-102/MC3/ALC-0315 for research only FDA approved Ideal delivery model Fluorescent liposomes Biological tracer Visual distinction LNPs with target groups Active targeting delivery Tissue/cell selectivity High bioavailability
  • 4.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 Lentiviral vectors (LVs) Integrating Stable expression Long-term expression Retroviral vectors (RVs) Integrating Transduce dividing cells Adeno-associated viral vectors (AAVs) Non-integrating Tissue tropisms Various mutants Low immunogenicity High level and long-term
  • 5.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
  • 6.Contact Us Phone: 1-631-624-4882 (USA) / 44-161-818-6441 (Europe) / Fax:1-631-938-8221 E-mail: / 45-1 Ramsey Road, Shirley, NY 11967, USA