Advances in Gene Therapy: MiniTEM™ Represents a Paradigm Shift in the Characterization of Viral Vectors

Gene therapy is one of the most progressing fields in life sciences. Although only eight gene therapy products have reached market approval, the pipeline is robust with ~500 candidates in different stages of clinical development. More than 1,700 clinical studies are being conducted, and the value of this industry segment is expected to grow by ~17% annually over the next 10 years.

Gene therapy involves using genetic material to fight or prevent disease. The gene is delivered into a patient’s cell using a vector. The most common types of vectors used in gene therapy are viruses. Viruses in their natural state make use of the host’s cellular machinery to synthesize proteins vital for the assembly of new functional viruses capable of replicating. The process of turning viruses into vectors involves removing all virulent genes and replacing them with a functional therapeutic gene, along with regulatory sequences to control its expression. Those modified viruses safely carry the inserted therapeutic gene into target cells with high efficiency.

Adenoviruses have been used for this purpose for over 20 years. More recently, adeno-associated viruses (AAVs) have become frequently used. For instance, AAVs are used in the first approved gene therapy drug in the Western world. In this report, we focus on AAVs and adenoviruses, but it is worth mentioning that other viruses such as lentivirus and retrovirus are also commonly used and have shown promise in clinical trials.

This report focuses on viral vector characterization and presents how highly automated and innovative analytical solutions from Vironova can perform detailed sample characterization quickly.

Find out how to automatically reveal and quantify the following undesired process outcomes.

  • Aggregation of viral particles
  • Failure to remove host-cell debris
  • Loss of viral particle morphology or integrity
  • Failure in the packaging of genetic material into the viral particles.

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