The Power Of A Multi-Vector Perspective: Nurturing LVV And AAV Development And Manufacturing Expertise In Parallel To Drive New Cycles Of Innovation

By Susan D’Costa, Chief Technology Officer, and Pratima Cherukuri, Chief Science Officer, Genezen

Over two dozen gene and gene-modified cell therapies have been approved for clinical use worldwide, and 50–75 cell and gene therapies are predicted to be on the market by 2030 in the United States alone. The growing rate of approvals is increasing the industry’s predicted compound annual growth rate; gene therapies are expected to account for a greater percentage of the market in the near future, thus creating a rising demand for adeno-associated viral (AAV) vectors.

While this growing number of approvals holds promise for enabling revolutionary treatments, there are still numerous manufacturing challenges that must be addressed to enable widespread adoption of these advanced therapies. Most gene therapies today are delivered using viral vectors, particularly AAVs, LVVs, and RVVs. Viral vector manufacturing requires significant financial investment to fund raw materials sourcing, specialized equipment, intricate processes, and rigorous quality assurance measures. This cost structure not only impacts the ultimate affordability of gene therapies but also the scalability and accessibility of treatments for patients.

Improving the productivity and efficiency of transient transfection processes for the manufacturing of AAVs at larger scale and addressing the need to cost-effectively produce large quantities of these complex molecules is essential to expand access to these life-saving treatments. Finding a CDMO partner with the resources and expertise to achieve efficient and scalable manufacturing is a key step in achieving the scalable manufacture of safe, effective AAV gene therapies.