When you first meet her, you won’t be able to tell that Ipek Kuzu suffers from a rare genetic disease. The three-year-old plays happily on her own for hours, driving her toy cars and “cooking” in her pretend kitchen. But she’s not well. She’s a little wobbly on her feet and doesn’t say much, and if nothing is done, she may die by her mid-20s. Ipek has ataxia-telangiectasia, or A-T, a disease caused by an error in her DNA. It causes the loss of brain cells, along with a high risk of infection and cancer.
top of page
Who We Are
Search
Zachary Brennan (Endpoints News) February 27, 2027 The FDA will increasingly use accelerated approval for gene therapies aimed at rare conditions, according to Peter Marks, the FDA’s top official over
Demaris Mills (Forbes) January 12, 2024 The 2023 Oligonucleotide Therapeutics Society meeting drew 800 attendees to Barcelona, a clear sign that nucleic acid-based drugs are becoming mainstream, espec
bottom of page