New Tumor Cases Confirm Cancer Risks of AAV Gene Therapies
A series of clinical tumor cases confirmed that adeno-associated viruses used in gene therapy can trigger oncogenesis, shifting safety evaluations.

A series of tumor cases reported this year provided convincing evidence that adeno-associated viruses (AAVs) used to deliver gene therapies can cause cancer in certain cases, according to ua.news citing STAT News ua.news
ua.newsua.newsOpen source ↗. For two decades, the potential of these small viral vectors to promote oncogenesis remained a subject of scientific debate and was regarded as largely theoretical. To date, AAV-based vectors have been administered to thousands of patients worldwide.
The documented cases have concluded discussions regarding whether such insertional risks exist in human applications. The confirmed oncogenic risk now forces clinicians, researchers, and patients' families to reassess the balance between potential adverse effects and the life-saving efficacy of genetic treatments.
According to University of Rochester neurologist Sam McKenzie, this development shifts the balance of considerations for certain patients with spinal muscular atrophy. Furthermore, researchers anticipate that analyzing these clinical cases will help establish technical parameters to improve the design and safety profile of future viral delivery platforms.



