4 results

• Skysona Blood Cancer Side Effects Lead to New FDA Restrictions

  August 11, 2025

  Martha Garcia

  The FDA has added a black box warning to the gene therapy drug Skysona after discovering more patients developed blood cancer than previously believed.

• Skysona Blood Cancer Risks Result in FDA Investigation Into Gene Therapy Treatment

  December 2, 2024

  Martha Garcia

  Federal health officials are investigating a potential link between the gene therapy Skysona and blood cancers, after clinical trials identified several patients who developed serious side effects in the months following treatment, including blood cancers and leukemia.

• Controversial Crispr Gene-Editing Can Damage Embryo DNA: Study

  November 5, 2020

  Martha Garcia

  A new study warns that Crispr gene-editing technology can cause potentially irreparable genetic damage and mutations.

• Data Used To Approve Zolgensma Gene Therapy Treatment May Have Been Manipulated: FDA

  August 7, 2019

  Russell Maas

  The FDA says a Novartis subsidiary manipulated data in animal trials used to approve a gene therapy treatment for children, but will let the treatment, the most expensive in the world, stay on the market due to a lack of other treatment options.