Despite an increasing number of gene therapy drugs in development and clinical trials every year, this industry is still in its early stages. While this evolving industry is able to benefit from the experience already gained in the field of recombinant antibodies, manufacturers today are facing new issues and challenges throughout the drug marketing journey – from development and manufacture, right through to the regulatory approval of AAV-based therapies.
Plan for Speed in AAV Development
In process development, speed to market and costs are critical considerations. Selecting the most suitable production system that results in fast production, high yields, and a high ratio of full-to-empty AAV capsids is key. Several producers are currently entering the market using transfection and adherent cells because it presents a fast route to market. There are however limitations to transfection-based manufacture, as typical AAV titers currently reach plateau at around 103-105 viral genomes per cell. Where higher yields are needed, alternative approaches are required. Developing suspension cultures or using stable producer cell lines can deliver long-term benefits for yield, scalability and cost but the additional development required may extend the time to market.
The viral vector manufacturing capacity is estimated to be 1 – 2 orders of magnitude lower than what is needed to support commercial supply requirements both today, and in the future. All parts of the industry are therefore focused on what is needed to achieve sustainable increases in capacity.
Learn more about the Pall products and services available for development and manufacturing of AAV based therapies.