Overcoming Obstacles In Gene Therapy Manufacturing

By John Kerwin and Patrick Starremans, Resilience

The gene therapy and viral vector space has yet to achieve the level of standardization that typifies established modalities, such as monoclonal antibodies. As a consequence, there exists a tremendous amount of diversity among these products. That diversity demands a complex and bespoke Chemistry, Manufacturing, and Controls (CMC) strategy, which creates a paradigm in which a manufacturer must establish an individualized approach for each product brought online. This in itself creates inefficiencies and delays that are often compounded by the relative dearth of expertise and experience among contract manufacturing and development organizations (CDMOs) new to these modalities.

The nascence of these products in general has resulted in bottlenecks around the building blocks that form the foundation of other more established therapeutics. Researchers across the space continue to work on defining the true critical quality attributes (CQAs) for these drug products, as well as the process parameters and testing methods best positioned to measure and monitor those attributes. As players across the landscape continue to develop the fundamental knowledge necessary to furthering these modalities, manufacturers with existing experience and investment in the variables that most impact scale and commercial viability will prove invaluable to biopharmaceutical companies. By partnering with a manufacturer that can perform end-to-end process and analytical development and cGMP manufacturing, companies can realize cost savings, establish an interoperable digital infrastructure, and optimize their CMC strategy.