Drugs / Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene

Follow last change Oct 2024 re-read 3 minutes ago

Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene

Developed for: mucopolysaccharidosis type 3A
Investigated by: CTI Clinical Trial and Consulting Services · Great Ormond Street Hospital for Children NHS Foundation Trust · Manchester University NHS Foundation Trust · Orchard Therapeutics · University College, London · University of Manchester

today NCT04201405 · Ph 1/2

2020202120222023202420252026

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 1/2	NCT04201405	Jan 2020 → Oct 2024 overdue	mucopolysaccharidosis type 3A	University of Manchester	Active not recruiting	No outcome recorded

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

Field	Value	Cited text
Known as	Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene	ClinicalTrials.gov intervention name — accepted as the source's own label NCT04201405 ↗
Modality	Cell therapy	“The investigational medicinal product (IMP) will be a cell-based gene therapy that uses genetically modified autologous CD34+ haematopoietic stem cells transduced with a...” NCT04201405 ↗
Target	SGSH	“a self-inactivating LV expressing the SGSH gene codon optimized for human use” NCT04201405 ↗