Cell and Gene Therapy: Transforming Treatment Paradigms for Patient-Centric Care

Abstract

Cell and gene therapies (CGTs) are transforming medicine by offering potential cures for diseases previously considered untreatable. Despite rapid advancements, challenges remain in optimizing efficacy and safety and ensuring patient accessibility and preference due to high costs and clinical uncertainties, particularly for rare diseases and one-time administration. The American Society of Clinical Pharmacology & Therapeutics (ASCPT) held a CGT satellite conference in 2025, titled "Cell and Gene Therapy: Transforming Treatment Paradigms for Patient-Centric Care." This manuscript summarizes the conference, covering gene therapies and T-cell immunotherapies from scientific, clinical, and patient-centered perspectives. Key topics on gene therapy included "platformization" to streamline development, lessons from adeno-associated virus-based gene therapies for hemophilia from patient and clinical perspectives, clinical pharmacology, and model-informed drug development (MIDD) considerations. The conference also highlighted T-cell immunotherapies including chimeric antigen receptor T therapy (CAR T), focusing on factors affecting cellular kinetics, efficacy, and safety, as well as emerging allogeneic CAR T for autoimmune diseases and MIDD strategies to optimize therapy design and clinical outcomes.

Keywords: cell‐ and tissue‐based therapy; gene editing; genetic therapy; immunotherapy; model informed drug development.

FIGURE 1

Clinical pharmacology principles in gene therapies. Exposure delivered to target cells drives transgene expression, leading to modulation of downstream biological pathways and therapeutic effects. Variability at each step can arise from product, patient, or disease‐related factors. (Conceptual framework adapted from Ref. [20]).