Protein translation: The genetic information is translated into a messenger molecule (mRNA) in the cell nucleus. The information contained in the mRNA sequence represents the blueprint for the production of the protein inside the cell.



Blocking protein expression: The antisense molecule specifically binds to the mRNA and consequently blocks the process of translating the blueprint into a certain pathogenic factor. As a result, the cell no longer produces this protein.

Our Technology

Antisense drugs are a new generation of gene-silencing therapeutic agents with potential for targeted causal treatment of as yet incurable diseases.

The enormous progress made in the field of basic medical and genetic research has shown that cancer is caused by defects in gene regulation. The result is either an overproduction or an abnormal production of proteins. Both effects are associated with cancer and progressive tumor growth. Preventing the production of these pathogenic proteins means combating cancer disease directly at its roots.

This is exactly where antisense drugs come in. Simply spoken, antisense technology selectively targets the production of specific proteins causing the disease. Antisense drugs are short, chemically modified, single-stranded oligonucleotide molecules, which interfere in the process of protein production.  They do this by binding to the blueprint of a certain protein and specifically prevent its conversion to a pathogenic factor that can cause uncontrolled tumor growth, for example. In contrast to gene therapy, antisense drugs do not alter human genes or have any effect on human genetic information.

Antisense Pharma is the first biotech company in Germany to successfully initiate and continue clinical studies of antisense drugs. The lead product trabedersen (AP 12009) has entered the last clinical phase necessary for potential market approval.

 

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Mode of action of antisense drugs: The antisense molecule is an exact mirror image of the mRNA of a certain pathogenic protein. Once bound to the mRNA, antisense drugs effectively block the generation of the related protein (e.g. tumor factor).