learned that when the
let's recode the building blocks
our focus
If it’s life-altering and difficult, we’re all in
We’re inspired by the people who live with a serious, life-altering disease but don’t let it stop them. The ones who keep pushing — reaching toward a day when they can live their lives fully. Because people in these situations are the ones who need help the most. So, we dig in to find new ways to recode the cells, reimagine the outcomes and rewrite the stories. It’s a quest that never gets old.
Learn about Cerebral Adrenoleukodystrophy
CALD is caused by accumulation of very-long chain fatty acids.
Learn about Transfusion-Dependent β-Thalassemia
TDT is characterized by reduced or absent functional β-globin.
