The Rise and Promise of Cell and Gene Therapy Hrvatska

Cell and Gene Therapy (CGT) is a cutting-edge medical approach encompassing both gene and cell therapy. Gene therapy aims to cure diseases by adding, modifying, or silencing individual genes' expression or repairing abnormal genes. Cell therapy, on the other hand, utilizes bioengineering methods to obtain cells with specific functions, which are then expanded and specially cultured in vitro to enhance their immune capabilities, kill pathogens and tumor cells, and thereby achieve the effect of treating certain diseases.

Compared to traditional small molecule and antibody drugs, CGT has the advantage of overcoming the limitations of protein-level regulation, enabling the treatment of rare diseases through gene expression, silencing, or vitro modification at the molecular level. Over 20 CGT products have been approved for marketing, primarily used to treat diseases such as diffuse large B-cell lymphoma, B-cell acute lymphocytic leukemia, mantle cell lymphoma, and multiple myeloma.

In the future, the application scope of CGT products will become even broader, with anticipated developments for the treatment of a series of diseases including HIV infection, cancer, hematological diseases, neurological diseases, and genetic diseases. However, the preparation process of CGT products is relatively complex, with plasmid production and virus production being critical steps.

During the preparation process, the first step is to obtain normal genes and construct them into vectors. Although amplifying the target fragment using a normal genome as a template is a common approach, it may encounter challenges such as difficult-to-amplify sequences and mutations in amplification products. Therefore, most researchers or companies opt for gene synthesis to directly obtain a constructed normal gene vector, significantly shortening the drug development and production cycle.

Yaohai Bio-Pharma can provide one-stop services for gene synthesis and vector construction, from sequence to plasmid. Our decade of extensive experience and over a hundred projects have enabled us to better understand and satisfy customer needs.

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