Phase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.
Therapeutic Candidate or Device Autologous Human CD34+ HSC from Mobilized PBSC of Patients with Cystinosis Modified by Ex Vivo Transduction using the pCCL-CTNS Lentiviral Vector Indication Cystinosis - An autosomal…