News & Event

Abstract BACKGROUND Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9 gene editing of the erythroid-specific enhancer region of BCL11A in autologous CD34+ hematopoietic stem and progenitor cells (HSPCs). METHODS We conducted

GENE-EDITING TREATMENT APPROVED BY HEALTH CANADA FOR SICKLE CELL DISEASE AND THALASSEMIA WAS TESTED IN ST. PAUL’S HOSPITAL CLINICAL TRIAL A gene editing treatment for sickle-cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) has received marketing authorization from Health Canada, following the results of a first-in-Canada

#SaveTheDate – The 1st TIF Pan American Conference on Thalassaemia & Other Haemoglobin Disorders & the 2nd RAIN Summit are coming to Washington D.C.! 🗓️ 11 – 13 July 2025 📍 Hyatt Regency Crystal City, Washington D.C., US 🗣️ In-person attendance only This landmark event,

Thalassemia is a genetic blood disorder that affects millions worldwide, including in Canada...