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Gene, Cell, & RNA Therapy Landscape Q1 2025

May 16, 2025

Welcome to the first quarterly report of 2025! During the past quarter, one new therapy was approved for each of the gene, cell, and RNA categories. In the U.S., the cell-based gene therapy Encelto became the first and only approved treatment for the degenerative eye disease macular telangiectasia type 2. An siRNA therapy, Qfitlia, was approved for hemophilia A or B. In China, the country’s first mesenchymal stem cell therapy, Ruiboshen, was approved for steroid-refractory acute graft-versus-host disease. During this past quarter, an increase continued in the number of non-genetically modified cell therapy trials initiated for non-oncology indications.

Of the 27 trials initiated, 74 % were for non-oncology indications — 16% higher than the previous quarter. In the gene therapy pipeline, there were 79 trials initiated in Q1, of which 57% were for oncology indications — the highest oncology trial proportion for the past year. Among RNA therapies in the pipeline, mRNA and RNAi continued to be preferred research modalities.

Companies in Q1 announced 90 deals in financing, alliances, and acquisitions, a 20% decrease from 113 in the previous quarter. While alliances and financings were down, acquisitions saw a slight uptick from seven to nine. A total of 12 start-ups raised $304.3 million in Q1 seed and Series A funding, representing a 20% decrease in volume and 50% decrease in value compared with the previous quarter.