International – Convergence: Regulatory considerations in advancing gene and cellular therapies

When developing a regulatory submission for a gene or cellular therapy, it’s essential to keep the basics in mind, such as how to demonstrate the product’s identity, purity, and potency, Haroon Hashmi, PhD, told attendees at RAPS Convergence 2021.

“Even when we think about these complex technologies — new emerging technologies — certain regulatory principles always stay in place,” said Hashmi, senior vice president at Ziopharm Oncology, which has developed the Sleeping Beauty T-cell therapy platform. “When you think about the CMC [it’s] identity, purity, potency,” he added, referring to the common acronym for chemistry, manufacturing and controls.

Advanced therapy sponsors will need to convince regulators that they can identify their therapeutic product based on its unique properties, show where there is the potential for impurities to be introduced during the manufacturing process or the gene transfer, and assess product potency to demonstrate that it is consistent using the manufacturing process, Hashmi explained…