Novartis reports nine cancer cases in patients treated with Zolgensma gene therapy

Novartis disclosed that nine individuals with spinal muscular atrophy who received its gene therapy Zolgensma later developed tumors.

Two of the cases showed evidence that the adeno-associated virus vector used to deliver the therapeutic gene had integrated into the patients' DNA, a finding that raises safety questions.

The company said it has informed health authorities about each case and will work with regulators on next steps. Patient groups such as SMA Europe have called for clear information.

The risk of AAV integration and potential cancer has been noted in Zolgensma’s prescribing information and has been a longstanding concern in gene-therapy research.