Cell and Gene Therapies (CGT) are transforming not just how humans treat genetic and intractable diseases but are altering the entire pharmaceutical ecosystem. CGT are rapidly becoming established as the new wave of biological therapeutics with autologous or allogeneic engineered cells as the active therapeutic (Cell Therapy), or the gene delivery system (Gene Therapy; e.g., LNP’s, AAV’s, etc.). More than 18 CTx or GTx products were approved by US FDA in 2020. Around 990 companies are engaged in R&D and commercialization of next-generation CGT. In short, CGT is in the process of becoming a mainstream.

Compared to other biological such as mAbs, BsAbs and proteins, CGT therapeutics are much more complex both physically and biologically, and present specific challenges for developing in vitro potency assays due to their complex mechanisms of action, complicated manufacturing processes and variable critical quality attributes (CQAs). This session will explore considerations and case studies demonstrating strategies of dealing with these biological complexities throughout the product life cycle.

Speakers:

Matthew Klinker, CBER, FDA

Potency Bioassays for Cell and Gene Therapy Products

Jen Fox, AstraZeneca

Development of Flow Cytometry-Based Methods for Release and Characterization of CAR-T Products

Serena Hamada, Bristol-Myers Squibb Company

Development of a Cytotoxicity Method for the Measurement of CAR-T Drug Product Killing Activity

Dan Lee, Novartis

Versatile Potency Assay for AAV Gene Therapy Drug Product

Michael Sadick

Appearances