The third quarter of 2025 brought notable progress across the gene, cell, and RNA therapy landscape, marked by four new global approvals — one in each major therapeutic category. In gene therapy, China’s NMPA approved Hrain Biotechnology’s Hicara CAR-T therapy for large B-cell lymphoma, while in the US, the FDA approved Precigen’s Papzimeos for recurrent respiratory papillomatosis. The FDA also granted approval for Ionis Pharmaceuticals’ Dawnzera, an RNA therapy for hereditary angioedema. In Europe, ExCellThera’s Zemcelpro, a non-genetically modified cell therapy for hematological malignancies, received authorization.

While regulatory progress continued, clinical development activity slowed, with 125 trials initiated across gene, cell, and RNA therapies. The global pipeline remains robust with more than 3,200 trials currently underway worldwide. Dealmaking activity increased in Q3, with 99 transactions representing a 9% rise from the previous quarter. This growth was supported by a rebound in start-up financing, totaling $230.9 million across 11 rounds. The quarter featured strategic takeovers by Kite and Biogen in areas of in vivo CAR therapies and ASO delivery, underscoring ongoing innovation and investment in next-generation modalities.

Pancreatic cancer joined the list of top five rare diseases targeted by CGTs. Additionally, the share of gene therapy trials focused on non-oncology indications rose to 42%, signaling a notable reversal of the downward trend observed since Q4 2024.

Across these indicators, the data tell a powerful story: progress in CGT is dynamic, but not linear. Each data point helps us better understand the pace and direction of the field, equipping the community to anticipate shifts and opportunities for impact. The data are critical to driving awareness, guiding research, and accelerating availability of cell and gene therapies for patients worldwide.

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