top of page
Menu
Close
HOME
ABOUT US
ABOUT PBCs
OUR APPROACH
THERAPIES & PIPELINE
RELATED NEWS
CONTACT US
RARITY PBC
NEWS & ARTICLES
Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
Donald Kohn awarded $14.7M CIRM grant to advance ADA-SCID gene therapy toward FDA approval
Autologous Ex Vivo Lentiviral
Gene Therapy for Adenosine Deaminase Deficiency
Lunch and Learn: ADA-SCID Gene Therapy Update
Gene therapy for ADA-SCID shows promise
Donald Kohn awarded $14.7M CIRM grant to advance ADA-SCID gene therapy toward FDA approval
5-year old with rare disorder awaits gene therapy treatment
bottom of page
