drugset
Drugs / CD11b.IDS-ApoEII
last change Jun 2023 re-read 3 minutes ago

CD11b.IDS-ApoEII

also known as CD11b.IDS-ApoEII Lentiviral Vector

Cell therapy targets IDS via restoration

Developed for
mucopolysaccharidosis type 2
Investigated by
CTI Clinical Trial and Consulting Services · Great Ormond Street Hospital for Children NHS Foundation Trust · LifeArc · Manchester University NHS Foundation Trust · University of Manchester

Trials 1

today NCT05665166 · Ph 1/2
20242025202620272028
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT05665166 Jun 2023 → Jul 2028 expected mucopolysaccharidosis type 2 University of Manchester Active not recruiting No outcome recorded

Evidence & citations 6 cited values

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

FieldValueCited text
Known as CD11b.IDS-ApoEII “Autologous CD34+ haematopoietic stem cells from MPS II patients will be genetically modified ex vivo using CD11b.IDS-ApoEII Lentiviral Vector (LV), a self-inactivating (SIN) LV...” NCT05665166 ↗
Known as CD11b.IDS-ApoEII Lentiviral Vector “Autologous CD34+ haematopoietic stem cells from MPS II patients will be genetically modified ex vivo using CD11b.IDS-ApoEII Lentiviral Vector (LV), a self-inactivating (SIN) LV...” NCT05665166 ↗
Action Restore “A working copy of the IDS gene is then placed into these cells in the laboratory (ex vivo).” NCT05665166 ↗
Modality Cell therapy “cell-based gene therapy” NCT05665166 ↗
Route Intravenous “given back to the child via a blood infusion” NCT05665166 ↗
Target IDS “expressing the human codon-optimized IDS gene” NCT05665166 ↗