Gene Therapy Studies Rose While Financing Declined In Q2 2025

In Q2 2025, progress in advanced therapeutics was marked by three notable approvals and continued growth across the gene therapy landscape. Pipeline activity also strengthened, with gene therapy programs increasing across all stages of development, from preclinical to Phase III. Oncology and rare diseases remain dominant areas, with oncological conditions accounting for eight of the ten leading rare disease targets. Among the 80 gene therapy trials launched in Q2, 64% focused on oncology—the highest share in the past year. Non-genetically modified cell therapies also advanced, though development leaned toward non-oncology indications; 76% of the 33 new trials in Q2 addressed conditions outside cancer. Meanwhile, RNA therapies saw a modest rise, with 38 new trials initiated—74% of which targeted non-oncology diseases. Together, these approvals and pipeline expansions underscore the momentum of gene, cell, and RNA therapies in addressing both rare and widespread conditions.