In the complex process of producing a gene therapy, bioprocessors depend on assays to monitor the attributes of the product at various stages. Scientists at Thermo Fisher Scientific focus on continually expanding and improving the available assays that bioprocessors can use.
One of these assays applies to bioprocesses that use the HEK293 human embryonic kidney cell line, which is often used in the development of a gene therapy. Thermo Fisher’s residual HEK293 qPCR assay uses a digital polymerase chain reaction to detect any host-cell DNA that remains in a gene therapy. Thus, this assay can be used to monitor the purity of a gene therapy product.
“In developing the HEK293 qPCR assay, a target with broad chromosomal coverage was essential, and we selected a gene distributed across multiple chromosomes to enhance sensitivity,” says Despina Lymperopoulou, PhD, senior manager, pharma analytics at Thermo Fisher Scientific. “Given HEK293’s human origin, minimizing background signal was a priority and was achieved through stringent manufacturing processes while maintaining assay sensitivity.”
Many gene therapies rely on an adeno-associated virus (AAV) to deliver a therapeutic gene. To help quantify that delivery, Thermo Fisher developed its AAV genomic titer dPCR assay.
Still, the company plans even more AAV-related assays that will improve the production of gene therapies. For example, “assays that measure full/empty capsid ratios and full-length genome integrity will be essential for ensuring the quality and efficacy of AAV-based gene therapies, as they help confirm that the therapeutic vector contains the correct genetic material,” says Lymperopoulou. “The cost of a full-scale AAV production run can range from $500,000 to over $1 million, depending on the scale and complexity of the production process, so protecting this expenditure will be essential to lowering cost in the future.”
To make gene therapies available to more patients, the entire production process must become more efficient, and improved assays will play a key role in reaching that objective.
The post Assays Improve Gene Therapy Production appeared first on GEN - Genetic Engineering and Biotechnology News.
One of these assays applies to bioprocesses that use the HEK293 human embryonic kidney cell line, which is often used in the development of a gene therapy. Thermo Fisher’s residual HEK293 qPCR assay uses a digital polymerase chain reaction to detect any host-cell DNA that remains in a gene therapy. Thus, this assay can be used to monitor the purity of a gene therapy product.
“In developing the HEK293 qPCR assay, a target with broad chromosomal coverage was essential, and we selected a gene distributed across multiple chromosomes to enhance sensitivity,” says Despina Lymperopoulou, PhD, senior manager, pharma analytics at Thermo Fisher Scientific. “Given HEK293’s human origin, minimizing background signal was a priority and was achieved through stringent manufacturing processes while maintaining assay sensitivity.”
Many gene therapies rely on an adeno-associated virus (AAV) to deliver a therapeutic gene. To help quantify that delivery, Thermo Fisher developed its AAV genomic titer dPCR assay.
Still, the company plans even more AAV-related assays that will improve the production of gene therapies. For example, “assays that measure full/empty capsid ratios and full-length genome integrity will be essential for ensuring the quality and efficacy of AAV-based gene therapies, as they help confirm that the therapeutic vector contains the correct genetic material,” says Lymperopoulou. “The cost of a full-scale AAV production run can range from $500,000 to over $1 million, depending on the scale and complexity of the production process, so protecting this expenditure will be essential to lowering cost in the future.”
To make gene therapies available to more patients, the entire production process must become more efficient, and improved assays will play a key role in reaching that objective.
The post Assays Improve Gene Therapy Production appeared first on GEN - Genetic Engineering and Biotechnology News.