Gene therapy approaches are increasingly being used for treating life-threatening diseases in humans. GeneWerk GmbH, a spin-off of the DKFZ and the NCT in Heidelberg, offers customised, high-resolution molecular and bioinformatic analyses that ensure the efficacy and safety of gene therapy and immunotherapy studies.
Gene therapy is considered one of the great beacons of hope for the treatment of incurable diseases. Viral vectors are already frequently used for transferring genes into human cells to treat life-threatening genetic defects. The number of clinical gene therapy trials worldwide is growing rapidly. Therapeutic results are extremely promising. However, in order to exclude severe adverse effects caused by gene therapy, viral gene shuttles need to undergo safety checks and resulting modifications in the cells need to be analysed. GeneWerk GmbH, a spin-off of the German Cancer Research Center (DKFZ) and the National Center for Tumour Diseases (NCT) based in the Heidelberg Technology Park, has unique expertise in determining the safety of viral gene therapy vectors. The company uses highly-efficient molecular analysis systems.
GeneWerk GmbH was founded by Prof. Dr. Christof von Kalle, Dr. Manfred Schmidt and Dr. Annette Deichmann in July 2014. Schmidt and Deichmann are the owners and directors of the company, in which the DKFZ is also directly involved. Oncologist and haematologist Christof von Kalle, director of the Department of Translational Oncology at the NCT and DKFZ and managing director of the NCT, is an internationally renowned scientist in the fields of stem cell research, mutation analysis and gene transfer. He is closely associated with the company as advisor.
Before molecular biologist Manfred Schmidt was involved in founding the company, he had already made a name for himself mainly through the further development of complex PCR technologies into analytical tools. Such tools can be used to check the insertion of viral DNA into the host genome with the utmost precision, right down to single-cell level. As head of the Molecular and Gene Therapy research group in the Department of Translational Oncology at NCT and DKFZ, Schmidt was involved in the most successful gene therapy studies in the world aimed at treating immunodeficiencies. Schmidt's role in the studies was monitoring the clonal composition of blood-forming cells, as knowledge of the expansion of a specific cellular clone provides information on the risk for later haematopoietic cancers.
Biologist Annette Deichmann has been involved in research in the fields of microbiology, biochemistry and human genetics at universities in Germany and abroad. She had some experience in industrial research before becoming involved in scientific project management as coordinator in von Kalle’s research department. She is still involved in the scientific side of these research projects.
GeneWerk’s team has many years of experience in integration-site analysis, high-throughput sequencing, whole-genome sequencing and bioinformatics, and caters for its customers' needs with a broad range of highly sensitive molecular methods. The company motto is “Precision biology for humans”, and it develops innovative research concepts and analysis platforms to ensure efficacy and safety in new gene therapy and immunotherapy studies. The creativity of the company’s own technological developments is apparent, for example, in what is known as “non-restrictive linear amplification-mediated PCR” (nrLAM-PCR). This technique provides a way to precisely analyse the insertion sites of viruses that are integrated as vectors in human cells, thus counteracting the risk of tumorigenesis induced by the vector systems.
In addition to nrLAM-PCR, GeneWerk also offers a method known as target enrichment sequencing (TES), which can also be used for amplifying sequences of interest.
The company also offers the following standard analyses:
Acquired data are subsequently analysed with bioinformatics methods by GeneWerk’s highly qualified staff according to clients’ individual requirements. High quality standards and the continuous optimisation of the analytical processes make it possible to deliver reproducible data within a relatively short time. The company is currently working on a new IT infrastructure in order to fulfil client requirements and efficiently evaluate the huge amounts of data. In addition, company certification is also on the cards. This upcoming certification and the company's broad range of validated analysis services mean that GeneWerk is also targeting the customers of the major international biopharmaceutical companies. Such potential customers are increasingly involved in gene therapy as a promising option for treating cancer and other incurable diseases.
Christof von Kalle, Annette Deichmann, Manfred Schmidt: Vector integration and tumorigenesis. Human Gene Therapy, June 2014, DOI: 10.1089/hum.2014.2525