Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

Authors
Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2021
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
Epistemonikos ID: ef8b8961e14847521b0dbb86dc53af4080c8ef7a
First added on: May 08, 2024