The Austrian contract research organization JSW acquired specific experience during the last couple of years in performing bio-distribution and safety studies for gene therapy programs. The company has developed and fully validated highly sensitive quantitative polymerase chain reaction procedures (qPCR) for detection of extremely small quantities of the gene of interest in different tissues and body fluids. For safety reasons it is important to guarantee extremely low detection thresholds either to quantify the distribution of the recombinant DNA through the whole organism or to ensure that organs and tissues different from the target tissue/organ do not carry any contamination of the gene of interest which has been transferred using the viral vector system.
For optimal treatment success with a high safety margin the expression of the gene of interest should take place in specified organs over specified time periods. The safety studies performed at JSW are defining not only the level of transgene copy number in different organ systems but also provide knowledge about transgene traceability in the course of time. The challenge is to guarantee lowest threshold levels of DNA detection to validate negative findings. In other words it must be guaranteed that finding no gene of interest really means that this tissue/organ is free of any contamination.
JSW’s staff invested enormous efforts into the development of these procedures in compliance with Good Laboratory Practice also considering the requested biological safety levels.
JSW developed a bio-distribution and safety screening for Amsterdam Molecular Therapeutics (AMT B.V., Amsterdam, The Netherlands) for their AAV-1 based gene therapy program. Investigations about bio-distribution of the therapeutic gene vector in mice were conducted at JSW. During the clinical test phase JSW routinely investigates now different human body fluids (serum, saliva, urine and sperm) to monitor the distribution and elimination of the viral vector in different organ systems.
Such careful and responsible investigations should help to define optimal safety for patients undergoing gene therapy in the future. For many patients, specifically those with congenital genetic defects, gene therapy offers great hope helping them to achieve optimal quality of life.
JSW is proud to be able to support these important therapeutic developments by safety monitoring on highest level of achievable quality.
For optimal treatment success with a high safety margin the expression of the gene of interest should take place in specified organs over specified time periods. The safety studies performed at JSW are defining not only the level of transgene copy number in different organ systems but also provide knowledge about transgene traceability in the course of time. The challenge is to guarantee lowest threshold levels of DNA detection to validate negative findings. In other words it must be guaranteed that finding no gene of interest really means that this tissue/organ is free of any contamination.
JSW’s staff invested enormous efforts into the development of these procedures in compliance with Good Laboratory Practice also considering the requested biological safety levels.
JSW developed a bio-distribution and safety screening for Amsterdam Molecular Therapeutics (AMT B.V., Amsterdam, The Netherlands) for their AAV-1 based gene therapy program. Investigations about bio-distribution of the therapeutic gene vector in mice were conducted at JSW. During the clinical test phase JSW routinely investigates now different human body fluids (serum, saliva, urine and sperm) to monitor the distribution and elimination of the viral vector in different organ systems.
Such careful and responsible investigations should help to define optimal safety for patients undergoing gene therapy in the future. For many patients, specifically those with congenital genetic defects, gene therapy offers great hope helping them to achieve optimal quality of life.
JSW is proud to be able to support these important therapeutic developments by safety monitoring on highest level of achievable quality.
