Elizabeth Csaszar1,3, Rohin Iyer1,2, Spencer Hoover1,3
1Centre for Advanced Therapeutic Cell Technologies, Toronto, ON, Canada
2General Electric Healthcare, Cell and Gene Therapy, Marlborough, MA, United States
3Centre for Commercialization of Regenerative Medicine (CCRM), Toronto, ON, Canada.
For many cell and gene therapy (CGT) companies, translating a potentially curative therapy from a new biological concept into a scalable and manufacturable product is the largest challenge to commercial success. Navigating this path is particularly difficult for the CGT field for multiple reasons: limited highly qualified personnel who have both the biological and process engineering know-how; an evolving regulatory strategy; and, investors with very aggressive timelines. Even for experienced teams, there is a difficult balance between achieving the major inflection point of the first clinical trial using a manual, open process, and building a more commercially suitable process along with the associated team and facility. Many companies are choosing to work with Contract Development and Manufacturing Organizations (CDMOs) to accelerate their clinical and commercial programs. In this article, we highlight key areas where CDMOs can help companies getting started in the world of CGT manufacturing, with considerations around timing of engagement to maximize commercial and clinical success.