Q2 2025 Gene, Cell, + RNA Therapy Landscape Report

Introduction

Welcome to 2025’s second quarterly report from ASGCT, Citeline, and Evaluate! This past quarter, three new therapeutics were approved, and the number of gene therapy programs continues to climb. In the US, FDA approved the cell-based gene therapy Zevaskyn for recessive dystrophic epidermolysis bullosa (RDEB) as well as the lower-dose mRNA vaccine mNexspike for COVID-19. In China, the country’s first gene therapy for hemophilia B was approved.

Gene therapy programs at each stage from preclinical through Phase III have continued to increase in Q2, with oncology and rare diseases remaining the top areas of development. Eight of the top 10 rare diseases in the overall pipeline are oncological, continuing a trend from the past three years. Of the 80 gene therapy trials initiated in Q2, 64% are for oncology indications, the highest proportion of the past year. In the cell therapy pipeline, oncology and rare diseases also remain the top areas of non-genetically modified cell therapy development. Of the 33 cell therapy trials initiated in Q2, 76% were for non-oncology indications. In the RNA therapy pipeline, 38 trials were initiated in Q2, up from 35 last quarter, of which 74% were for non-oncology indications.

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