Hematopoietic Stem/Progenitor Cell-Based Chimeric Antigen Receptor Gene Therapy for HIV Infection

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Grant Award Details

Grant Number:
Disease Focus:
Human Stem Cell Use:
Cell Line Generation:
Award Value:

Grant Application Details

Application Title:

Hematopoietic Stem/Progenitor Cell-Based Chimeric Antigen Receptor Gene Therapy for HIV Infection

Public Abstract:
Translational Candidate

A blood forming stem cell based therapy to treat HIV infection and enhance HIV immunity.

Area of Impact

We are seeking to develop a therapy to treat HIV infection to replace standard drug therapy and cure people of the virus.

Mechanism of Action

We are seeking to develop a gene therapy that modifies a HIV infected individual’s immune system to directly attack HIV infected cells in a better way than would occur naturally and also protect these modified cells from being infected themselves. Through the enhancement of these immune responses, we are attempting to provide a way for HIV to be cleared from the body.

Unmet Medical Need

HIV infection remains a top public health concern worldwide and, though it can be managed with therapy, there is no curative treatment that is available for all. An improved means to eradicate HIV in every infected individual is needed.

Project Objective

Our goal is to have pre-IND meeting with the FDA.

Major Proposed Activities

  • We will establish Good Manufacturing Practice (GMP) processes for gene therapy vector production and cell product manufacturing.
  • We will develop assays and perform preclinical safety and pharmacology studies to identify dosing and efficacy in humanized mice.
  • We will prepare clinical and regulatory protocols towards approval for further IND and clinical trial development.
Statement of Benefit to California:
California ranks second in the nation in cases of HIV, with over 170,000 persons currently living with HIV with the direct healthcare cost to California approaching $1.8 billion annually. A curative treatment is therefore a high priority. A stem cell based therapy offers promise for this goal, by providing an inexhaustible source of protected, HIV specific immune cells that would provide constant surveillance and potential eradication of the virus in the body.