drugset
Drugs / transduced CD34+ hematopoietic stem cell
last change Oct 2025 re-read 3 minutes ago

transduced CD34+ hematopoietic stem cell

targets ABCD1, ARSA

Developed for
adrenoleukodystrophy · metachromatic leukodystrophy
Investigated by
Guangzhou Women and Children's Medical Center · Shenzhen Second People's Hospital · Shenzhen University

Trials 1

today NCT02559830 · Ph 1/2
2015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT02559830 Jan 2015 → Oct 2025 adrenoleukodystrophy, metachromatic leukodystrophy Shenzhen Second People's Hospital Unknown 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 transduced CD34+ hematopoietic stem cell ClinicalTrials.gov intervention name — accepted as the source's own label NCT02559830 ↗
Target ABCD1 “arylsulfatase A（ARSA) / adenosine-triphosphate-binding cassette, sub-family D (ABCD1) gene transfer into hematopoietic stem/progenitor cells” NCT02559830 ↗
Target ARSA “arylsulfatase A（ARSA) / adenosine-triphosphate-binding cassette, sub-family D (ABCD1) gene transfer into hematopoietic stem/progenitor cells” NCT02559830 ↗