Gene Therapy clinical trials at UC Health
2 in progress, 1 open to new patients

• Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

  open to eligible males ages up to 5 years

  This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol. Enrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized. Harvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized

  at UCLA

• Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1

  Sorry, in progress, not accepting new patients

  Phase 3 pivotal US trial studying open-label intravenous administration of AVXS-101 in SMA Type 1 patients

  at UCLA

Last updated: April 3, 2019