The fourth quarter of 2025 marked continued regulatory progress across the gene, cell, and RNA therapy landscape, with three new approvals spanning gene and RNA modalities. In gene therapy, regulatory agencies in both China and the United States advanced novel treatments for rare and serious conditions. The FDA approved GSK’s Waskyra, a hematopoietic stem cell–based gene therapy for Wiskott-Aldrich syndrome, while China’s NMPA approved Chongqing Precision Biotech’s Pulidekai, a CD19 CAR-T therapy for relapsed or refractory acute lymphoblastic leukemia. In RNA therapeutics, the FDA also granted marketing approval to Arrowhead Pharmaceuticals’ Redemplo for familial chylomicronemia syndrome.

Despite these regulatory milestones, the overall development pipeline continued to contract modestly in Q4. The total number of gene, cell, and RNA therapies in development declined to 2,041, representing a 4% decrease from Q1 2025 levels. This shift was largely driven by a reduction in preclinical programs, which fell from 66% to 60% of the pipeline, reflecting a broader recalibration of early-stage portfolios rather than downstream attrition. Notably, the proportion of therapies in Phase I through preregistration remained relatively stable, underscoring continued commitment to advancing programs already in clinical development.

In contrast to the tightening pipeline, start-up financing activity strengthened further in Q4. Advanced molecular therapy companies raised $557.1 million across 14 transactions, representing a 141% increase in total value from the prior quarter. Dealmaking activity overall remained steady at 100 transactions, signaling sustained strategic interest amid a more selective development environment.

Taken together, Q4 2025 illustrates a field in transition — balancing near-term regulatory achievements, disciplined pipeline optimization, and renewed investor confidence as innovation continues to evolve across CGT modalities.

For the full report, please download the PDF version at the top of this page in collaboration with ASGCT.