Hematopoietic Stem Cell Gene Therapy for Wiskott Aldrich Syndrome

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Disease Focus:
Human Stem Cell Use:
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Grant Application Details

Application Title:

Hematopoietic Stem Cell Gene Therapy for Wiskott Aldrich Syndrome

Public Abstract:
Translational Candidate

Human hematopoietic stem cells that have been modified to express a functional WAS gene to treat patients with Wiskott Aldrich Syndrome (WAS)

Area of Impact

These studies will bring stem cell gene therapy for WAS closer to the clinic especially for those without an HLA match or disease too severe for HSCT

Mechanism of Action

Hematopoietic stem cells (HSCs) with defective WASp expression are modified with a lentiviral vector which restores a normal copy of the defective gene.
Transplantation of gene-modified HSCs, which are self-renewing and long-lived, produce all blood lineages, including white blood cells and platelets which can correct the severe autoimmunity, immunodeficiency, and bleeding episodes present in WAS

Unmet Medical Need

There is no curative treatment for WAS patients without a bone marrow match. Gene corrected HSC can cure WAS and provides a therapeutic option for these patients.
This proposal will advance the field of stem cell gene therapy and treatment of primary immune disorders.

Project Objective

Conduct a successful Pre-IND meeting

Major Proposed Activities

  • Obtain clinical grade lentiviral vector and demonstrate the ability to manufacture the stem cell product at clinical scale
  • Perform rodent studies to assess safety and the effective dosage of the cell product
  • Prepare Pre-IND package. Complete Pre-IND meeting with the FDA.
Statement of Benefit to California:
Safe, definitive therapies for Wiskott Aldrich Syndrome represent an unmet medical need. Allogeneic stem cell transplant is frequently complicated by graft-versus-host disease or limited by lack of HLA matched donors. Successful demonstration that stem cell gene therapy can safely and effectively cure WAS will shift the paradigm by which patients will be treated and provide a foundation by which other immune and blood diseases may be cured in the future.