Newsletter | April 25, 2023

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


Interested in strategies to streamline your viral vector process development and manufacturing scale up while reducing risk and costs? We invite you to utilize these technical resources.

Driving Down The Cost Of Goods For Viral Gene Therapies To Better Serve Patients

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.

Move Your Gene Therapy From Strategy To Reality

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.

Development And Scale-Up Of A Helper-Dependent Adenovirus (HDAd) Process In Adherent Cell Culture

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.

Minimizing Risk In AAV Applications
Considerations Of Quality By Design (QbD) Principles For Sterilizing-Grade Filters In AAV Applications

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.

Apply Quality By Design (QbD) Principles For Adeno-Associated Virus

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.

Industry Webinar
Successful Suspension-Based Viral Vector Manufacturing Scale-Up

As gene therapies move toward commercialization, the journey to manufacture a high-quality product while achieving economies of scale and reducing facility footprint can be challenging. Watch this panel webinar and hear from gene therapy thought leaders and experts from industry and academia, including Dr. Timothy Cripe (Nationwide Children’s Hospital) and Lenore Giannunzio (Andelyn Biosciences), as they discuss best practices to successful suspension-based viral vector manufacturing scale-up.