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Medicilon nucleic acid drug R&D platform
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2022-09-02
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Medicilon nucleic acid drug R&D platform is an integrated and comprehensive solution that covers drug discovery, CMC and preclinical research services. Based on a rigorous scientific attitude, an open-minded scientific spirit and advanced equipment, our integrated solution can meet the industry's research and development needs for cutting-edge innovative nucleic acid drugs, and undertake research programs like nucleic acid drug discovery, screening and preclinical research services for pharmaceutical companies and scientific research institutions.

Popular types

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Advantages

For its fast and intuitive design of base sequences, the development of nucleic acid drugs applies simple materials, convenient preparation processes and affordable production costs, which greatly shorten the drug development cycle, making it possible to customize individualized treatment plans. Hence, it offers a feasible solution for rare diseases and other problems currently plagued.

Nucleic Acid Synthesis and Chemical Modifications

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Bioactivity Screening

  ❖ Evaluation of binding between siRNA-GALNAc and targeted liver cells. (ELISA,SPR,FP,FACS, MSD, Confocal microscope)

  ❖ Evaluation of decrease in target mRNA/protein level

  ❖ Evaluation of cell phenotypes and functional interfere

  ❖ Evaluation of off-target effect

             Searching for potential off-target mRNA/protein in the database, such as NCBI, nucleotideBLAST

             Overall analysis applying RNAseq or RNA Microarray

Bioactivity Screening.jpg

Nucleic Acid Process R&D

※ Select starting materials
We aim to choose starting materials with traits like easy to purchase, mild toxicity, good quality stability;

※ Process R&D of nucleic acid
We aim to develop stable and green synthesis routes with low cost and high security.

※ Quality Control
Up-to-date quality control system with complete technical standard.

※ Scale up
End to end service ensuring smooth transfer.

Nucleic Acid Drugs Preparation

Due to their low immunogenicity, biocompatibility, and high encapsulation efficiency for oligonucleotide molecules, lipids and their derivatives have become the go-to delivery systems for nucleic acid drugs that have attracted much attention in recent years. The system is positively charged in the physiological environment. The negatively charged nucleic acid molecules are encapsulated by electrostatic action, and the positively charged surface can also help the entire carrier system to combine with the cell membrane of the target cell, thereby playing a delivery role.

Common Delivery Systems

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Medicilon's Preparation Methods of Nanoparticles

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Traits of Successful Delivery Systems

  ❖ Easily modified, easily synthesized, easily produced.

  ❖ The on-target and off-target ratio of delivery should be within an acceptable range.

  ❖ The effective dose must be significantly lower than the toxic dose.

  ❖ The bioactivity of the nucleic acid should be consistent from batch to batch.

  ❖ In most clinical cases, repeated administration does not result in loss of efficacy or safety.

Medicilon's Nanoparticle R&D Lab

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Nucleic Acid-Lipid System R&D

  ❖ Formulation: drug to lipid ratio, solvent screening, aqueous to organic solvent ratio

  ❖ Process: Preparation methods

  ❖ Stability

  ❖ Dosage form screening

    Nucleic Acid Drugs Bioanalysis

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    Solutions for Nucleic Acid Bioanalysis

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    Pharmacology evaluation

      ❖ Contrast of different drug delivery methods.

      ❖  Relevance analysis between pharmacology and target mRNA/protein degradation & nucleic acid drug PK

    Pharmacology evaluation.jpg

      ❖  Dendrimer LNP

      ❖  Luc mRNA; Ⅳ and intra-tumoral

    Nucleic acid drugs15.jpg

      ❖  CNP-generated exosome

      ❖  Glioma-directed exosomal Exo-T delivery of PTEN mRNA,IV

    Nucleic acid drugs16.jpg

      Contact Us
      E-mail:
      marketing@medicilon.com
      Address:
      585 Chuanda Rd, Pudong, Shanghai, China, 201299 (Headquarters)
      1 Broadway, 9th Fl, Cambridge Innovation Center, Cambridge, MA 02142, US
      Kelvinstraat 41b, 6601 HH Wijchen, The Netherlands
      Allia Future Business Centre Kings Hedges Road Cambridge CB4 2HY, UK

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