Drugs / Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
last change Jul 2022 re-read 3 minutes ago

Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a

Cell therapy

Developed for
sickle cell disease
Investigated by
Blood and Marrow Transplant Clinical Trials Network · California Institute for Regenerative Medicine (CIRM) · David Williams · Genetix Biotherapeutics Inc. · National Heart, Lung, and Blood Institute (NHLBI)

Trials 1

20232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 2 NCT05353647 Jul 2022 → Oct 2027 expected sickle cell disease David Williams Active not recruiting No outcome recorded

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a ClinicalTrials.gov intervention name — accepted as the source's own label NCT05353647 ↗
Modality Cell therapy “a single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a” NCT05353647 ↗
Route Intravenous “The transduced cells will be infused intravenously over 30-45 minutes” NCT05353647 ↗