04.25.23 -- Driving Down The Cost Of Goods For Viral Gene Therapies

Gene therapies for ultra-rare diseases will be commercially viable only if we drive costs down. There is a need for real innovation to help shift the paradigm of the cost per dose. In this article, Emmanuelle Cameau, strategic technology partnership leader, cell and gene therapy, Pall Corporation, discusses how a combination of optimizing your process development, and process intensification can help drive down the cost of doses.

Adeno-associated viral vectors are the most frequently used delivery system for gene therapies, and the need for more cost-effective and scalable viral-vector manufacturing platforms is growing. Explore how optimizing your process with an end-to-end integrated solution can reduce costs, increase speed-to-market, and get your final gene therapy product within reach of patients.

This poster presentation demonstrates the successful steps taken to develop and scale-up a process for a HDAd, leveraging Pall’s process expertise and the iCELLis® bioreactor platform to help accelerate the development timelines.

Sterilizing-grade filtration plays an important role in AAV manufacturing, both for drug substance (DS) as well as final drug product (DP) to ensure patient safety. Here, we reference a white paper that provides new guidance and outlines recommendations based on QbD principles for the successful implementation of sterilizing-grade filters in AAV manufacturing processes.

A key aspect of chemistry, manufacturing, and controls (CMC) documentation for complex biological products is the application of quality by design (QbD) principles: a rationale of quality being achieved by process design rather than relying on final quality testing alone. The work presented here provides a framework to illustrate the concept and initial thoughts on using QbD concepts for gene therapy, specifically adeno-associated virus (AAV) production.