4 New Approvals In Q3 Across Gene, Cell, RNA Therapies

Q3 2025 delivered meaningful momentum across the gene, cell, and RNA therapy landscape, highlighted by four global approvals. China’s NMPA cleared Hrain Biotechnology’s Hicara CAR-T for large B-cell lymphoma, the FDA approved Precigen’s Papzimeos for recurrent respiratory papillomatosis, and Ionis Pharmaceuticals secured FDA approval for Dawnzera, an RNA therapy for hereditary angioedema. In Europe, ExCellThera’s Zemcelpro, a non-genetically modified cell therapy for hematologic malignancies, also received authorization.

While regulatory milestones advanced, trial initiations slowed to 125 across the three modalities, though the global pipeline remains strong with more than 3,200 active studies. Dealmaking gained energy with 99 transactions — a 9% quarterly increase — supported by renewed start-up financing totaling $230.9 million. Strategic acquisitions by Kite and Biogen highlighted continued investment in in vivo CAR technologies and ASO delivery. Notable shifts in development focus emerged as pancreatic cancer entered the top five rare diseases targeted by CGTs, and gene therapy programs for non-oncology indications climbed to 42%, reversing a previous downturn.

Overall, the quarter reflected a dynamic but uneven trajectory — one that underscores the importance of data-driven insight to track progress, anticipate shifts, and advance transformative therapies for patients.