USA — Thermo Fisher Scientific has introduced the Gibco CTS AAV-MAX Helper-Free AAV Production System, a new all-in-one solution designed to help meet clinical and commercial demands for cost-effective and scalable development of adeno-associated virus (AAV)-based gene therapies.

It is currently the only product of its kind manufactured under cGMP conditions, allowing for large-scale applications.

AAV is used in 82% of viral vector-based gene therapies in development, and scaling up production is critical to accelerating the transition from research to commercialization and bringing gene therapies to market faster and at a lower cost.

The system can save manufacturers up to 50% on production costs when compared to alternative polyethyleneimine (PEI)-based suspension systems, and it can help reduce plasmid DNA costs by 25%.

It features a mammalian-based suspension system comprised of a clonal HEK293 cell line and animal origin-free reagents that support scalable AAV production from shake flask to bioreactor scale.

Building on 60 years of trusted Gibco solutions, the CTS AAV-MAX System is the latest addition to our fit-for-purpose line of scalable solutions for commercial manufacturing of cell and gene therapy applications,” said Amy Butler, president of biosciences, Thermo Fisher Scientific.

As part of Cell Therapy Systems (CTS) solutions, the CTS AAV-MAX System is designed to work within the AAV production workflow, from cell culture and transfection through AAV viral vector production.

The CTS AAV-MAX System allows researchers to seamlessly transition from research on the Gibco AAV-MAX Helper Free AAV Production System to clinical scale.

Meanwhile, researchers have created a family of AAVs that are more than three times more effective than the current leading AAV delivery vehicle, AAV9.

They used an mRNA-based directed-evolution strategy in multiple mouse strains to accomplish this.

Furthermore, in cynomolgus macaques, they used a de novo selection to identify engineered vectors with increased potency in the brain.

The new AAVs can pass through the blood-brain barrier, which normally prevents many drugs from entering the brain.

The engineered AAV capsids also accumulate much less in the liver than AAV9, potentially reducing the risk of liver side effects that have been seen in other AAV9-based gene therapies.

This family of AAVs, called the PAL family, could be a safer and more efficient way to deliver gene therapies to the brain.

This work is published in Med in the article, “Systemic administration of novel engineered AAV capsids facilitates enhanced transgene expression in the macaque CNS.”

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