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Gene Therapy R&D Solutions

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CD Formulation has extensive experience in providing gene therapy solutions to help our clients overcome potential obstacles and challenges. With our expertise, we can assist you in developing innovative treatments. From gene therapy research to commercialization, our comprehensive solutions, high-quality products, and exceptional service support you every step of the way.

Our Gene Therapy R&D Solutions

CD Formulation's gene therapy solutions integrate cutting-edge technology with specialized services tailored to deliver a comprehensive treatment strategy for our clients. Our team possesses extensive experience in the industry and is dedicated to offering scientific, customized services while fostering innovation and the application of gene therapy.

Nucleic Acid Customization

CD Formulation offers high-quality raw materials specifically designed to optimize the synthesis of small nucleic acids and mRNAs, facilitating your transition from development to production and commercialization. We connect you with partners who can assist you in achieving your nucleic acid therapy development objectives.

mRNA-based therapeutics and vaccines are produced through biosynthesis, utilizing nucleotides, enzymes, and mRNA modification systems.

Synthetic small nucleic acid therapeutics are produced through chemical synthesis utilizing DNA or RNA phosphoramidite monomers.

Gene Therapy R&D Solutions

Our goal is to support you at every stage of the development process for your gene therapy production solution. We can help you achieve your gene therapy objectives by offering regulatory support and filings, tailored gene therapy products and services, and comprehensive assistance.

The following is the process of gene therapy development.

Fig.1 Gene therapy development solutionsFig.1 Process of gene therapy development. (CD Formulation)

  • Plasmid Development and Production

Achieve faster process development with tools specifically designed to scale up production and to isolate, purify, and extract stable, high-quality plasmid DNA.

  • Cell Expansion

As a leading manufacturer of high-volume culture media, we can assist you in increasing productivity, minimizing supply risks, and enhancing efficiency.

  • Plasmid Transfection

For efficient gene transfer via transfection, plasmid DNA can be complexed with lipid reagents to facilitate effective delivery to the nucleus.

  • Viral Vector Development

Regardless of the production scale or cell line utilized, we can offer cutting-edge products for your upstream viral vector production workflow.

  • Formulation Development

We provide pharmaceutical filling, finished product, and storage solutions, including sterile container systems for therapeutic delivery.

  • Quality Control

We assist you in implementing our bioproduction solutions to ensure compliance with regulatory requirements.

Our Available Services and Platforms

Target Prediction and Validation Platforms

Delivery Vector Development Platforms

Efficacy Evaluation Services

Our Workflow of Gene Therapy R&D Solutions

Understanding Disease Mechanisms

Bioinformatics approaches offer new insights into disease processes by leveraging genomic information linked to various diseases. Target genes associated with specific conditions can be identified through sequencing, quantitative PCR (qPCR), and genome-wide screens utilizing CRISPR guide RNA (gRNA).

Target Gene Validation

To validate targets, researchers employ techniques such as cell-based modeling, phenotypic modeling, immunohistochemistry (IHC), fluorescence-activated cell sorting (FACS), and Western blotting (WB) to demonstrate target engagement and establish that the target is involved in the progression of a specific disease.

Vector Selection

According to the characteristics of the target gene and the therapeutic requirements, suitable gene vectors (such as viral or non-viral vectors) are chosen to ensure the effective delivery of the therapeutic gene to the target cells.

Gene Construction

The design and construction of expression vectors aim to combine the target gene with suitable promoters and regulatory elements, ensuring effective gene expression within the cell.

In Vitro Experiments

Conduct preliminary experiments using cellular models to evaluate the efficacy and safety of gene therapy, focusing on gene transduction efficiency and the restoration of cellular function.

Publication Data

Technology: Delivery of gene therapy drugs by viral vector and non-viral vector technologies

Journal: Asian journal of pharmaceutical sciences

IF: 10.2

Published: 2021

Results:

Currently, gene therapy drugs such as siRNA, shRNA, antisense oligonucleotide, CRISPR/Cas9 system, plasmid DNA and miRNA have shown great potential in biomedical field. This review mainly elucidates the widely used gene therapy drugs, summarizes the viral and non-viral vectors for delivering gene therapy drugs, and summarizes the application of gene therapy in the treatment of genetic diseases. In addition, the challenges and opportunities facing the field are explored from the perspective of developing effective nanodelivery systems.

Fig.2 Delivery of gene therapy drugs by viral vector and non-viral vector technologiesFig.2 Types of gene therapy drugs. (Pan X, et al., 2021)

CD Formulation offers a comprehensive range of gene therapy solutions, including nucleic acid therapy development solutions and gene therapy development solutions, designed to provide customers with innovative and efficient technological options. Our team of experts is committed to delivering cutting-edge research and development support tailored to your specific needs. For more information, please contact us.

References

  1. Pan X, Veroniaina H, Su N, et al. Applications and developments of gene therapy drug delivery systems for genetic diseases. ASIAN J PHARM SCI. 2021, 16(6): 687-703.
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At CD Formulation, we understand the unique challenges and opportunities associated with nucleic acid formulation development. Our team of experts is dedicated to providing tailored solutions...

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