Hematopoietic Stem Cell Gene Therapy for XCGD

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Grant Award Details

Grant Number:

Human Stem Cell Use:
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Grant Application Details

Application Title:

Hematopoietic Stem Cell Gene Therapy for XCGD

Public Abstract:
Therapeutic Candidate or Device

Hematopoietic stem and progenitor cells collected from X-CGD patients modified with a highly regulated lentiviral vector


X-linked Chronic Granulomatous Disease

Therapeutic Mechanism

Lentiviral vector (LV) modification of autologous hematopoietic stem and progenitor cells (HSPCs) to restore physiologic gp91phox expression. We have developed a next-generation LV designed by bioinformatic-guided screening of enhancer/promoter elements from the human CYBB gene to restore physiologically regulated expression of gp91phox protein.

Unmet Medical Need

Allogeneic transplant, while curative, is not available to patients without a matched donor, an issue that is particularly exacerbated for patients from ethnic minorities.

Project Objective

Submit an IND to initiate a Phase I/II trial

Major Proposed Activities

  • Complete CMC requirements (vector production and cell manufacturing)
  • Complete toxicology studies (in relevant mouse model and cell culture systems)
  • Initiate documentation required to open a PhaseI/II trial
Statement of Benefit to California:
This proposal uses the first product from our pipeline that creates highly regulated vectors. The resulting Phase I/II trial should demonstrate clinical superiority of our vector and provide a treatment for X-linked Chronic Granulomatous Disease. Moreover this trial will pave the way for other vectors in our pipeline to treat a range of primary immune deficiencies and other genetic blood diseases. This will result in improved patient care in the state of California and around the world.