Putting in place a QC-friendly potency assay to support commercialization of cell and gene therapy (CGT) products will require an iterative process that needs to be thought out early in development with regulator input, experts involved are advising.
The need for iterative CGT assay development supported by communications with the regulators emerged as a key theme during the panel discussion at the opening session of a CASSS meeting on the “scientific approaches and regulatory strategies” for bioassays, held in late March in Silver Spring, Maryland.
In recognition of the wave of cell and gene therapies that are entering the biopharma pipeline, the opening session at the meeting was focused specifically on the challenges of developing bioassays that are viable in measuring the potency of CGT products.
Presentations at the session were given by FDA and industry CGT experts, including: ● Center for Biologics Evaluation and Research (CBER) Office of Cell and Gene Therapies Reviewer Xiaobin (Victor) Lu ● Celladon’s Barbara Thorne ● Novartis’ Erik Rutjens, and ● Ocata Therapeutics’ Irina Klimanskaya. [An in-depth review of Lu’s remarks and more on the company presentations will follow in IPQ.]
The panel discussion held after the presentations was designed to compare participants’ experiences to date in the development and implementation of potency assays for CGTs, and to explore the challenges and where solutions may lie.
The panel included the speakers from the opening session, joined by Health Canada Center for Evaluation of Radiopharmaceuticals and Biotherapeutics Senior Regulatory Scientist Anthony Ridgway.
Along with challenges of developing QC-friendly potency assays that will support commercialization and the iterative process involved, the panel explored: ● developing reference standards ● setting specifications ● designing commercial control systems ● the cell lines being used and associated assays, and ● dosing and safety considerations.