At the European Cell & Gene Therapy Summit 2025, funding challenges and innovation drove discussions on the sector’s future.
Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased ...
Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.
News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects an ...
New study data showed treatment slowed signs of disease progression by 75% after three years, a finding that could have ...
UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback