How Can (Bio)pharmaceutical Companies Contribute to Improving Global Access to Essential Medications?

Posted: 7 August 2024

Industry experts were asked to weigh in on how companies can help to keep innovation and access open. Jason King, PhD, was featured in this list. In his section, he focused on how CDMOs can help to streamline and strengthen innovation in the cell and gene therapy sector:

The price, particularly of cell and gene therapy medicines, is currently too high, even in relatively wealthy healthcare systems. This can make the challenge of serving less affluent regions even more daunting.

CDMOs can do several things to generally reduce the per-dose cost of these new medicines. These include developing more effective scaled-up or scaled-out manufacturing processes to generate more product. This is already needed for some indications during the clinical trial phase where larger amounts of vector are required per patient but must continue to improve as these new therapies make it into the commercial phase.

In addition to the scaling considerations, CDMOs can look to improve the volumetric or per-cell productivity of viral vector manufacturing so that more patient doses can be generated from the same sized bioreactor. In AAV vector manufacturing, vector productivity depends heavily on both the size and nature of the therapeutic cassette and the capsid type doing the actual delivery. The introduction of new production platforms and process development and intensification with existing ones has greatly improved AAV productivity, which a few years ago was around 1011vg/mL in the bioreactor and is now edging closer to 1012vg/mL levels.

Focusing on disease indications that require the smallest active vector doses may help establish gene therapy workflows in low-income regions. Ophthalmic indications for example may only need about 5 x 1010 vg per eye, which can result in a lot more patients being treated per bioreactor run.