Grant Award Details
- A well-prepared pre-IND meeting with the FDA for a HIV-specific CAR-modified HSPC cell product to treat and eliminate chronic HIV infection.
Grant Application Details
- Hematopoietic Stem/Progenitor Cell-Based Chimeric Antigen Receptor Gene Therapy for HIV Infection
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.
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.
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.