Gene Therapy clinical trials at University of California Health

2 research studies open to eligible people

Showing trials for

  • Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma

    open to eligible people ages 18 years and up

    The goal of this clinical trial is to evaluate the safety, tolerability, and recommended Phase 2 Dose (RP2D) of KLN-1010 in patients with relapsed or refractory multiple myeloma.

    at UCSF

  • Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

    open to eligible males ages 0-5

    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

Our lead scientists for Gene Therapy research studies include .

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