Gene Transfer for Patients With Sickle Cell Disease

Phase 1/2 Terminated 7 enrolled Children's Hospital Medical Center, Cincinnati

NaSingle-groupOpen-labelSupportive care

Summary

The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease

Timeline

Start: 2014-07
Primary completion: 2022-10-31
Completion: 2022-10-31

Outcome

Met primary endpoint

Stopped (Business): “Sponsor decision due to funding”

paper the predetermined primary endpoints were met PMID 40419809 ↗

Publications

Grimley M, Davies SM, Shrestha A, Shova A, Asnani M, Kent M, Sayani F, Quinn CT, Niss O, Lutzko C, Mehta PA, Khandelwal P, Little C, Chandra S, Felker S, Chi M, Kalfa TA, Knight-Madden J, Arumugam PI, Ramos KN, Witting S, Latham T, Bushman FD, Malik P. Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial. Nat Med. 2025 Jul;31(7):2204-2212. doi: 10.1038/s41591-025-03662-2. Epub 2025 May 26.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	ARU-1801	Unknown	—	Intravenous