Grant Award Details

Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients
Grant Number: 
CLIN2-08289
Project Objective: 
  • To complete a Phase I clinical trial.

Investigator: 
Type: 
PI
Disease Focus: 
HIV/AIDS
Infectious Disease
Blood Cancer
Cancer
HIV-related Lymphoma
Human Stem Cell Use: 
Adult Stem Cell
Award Value: 
$8,414,265
Status: 
Active

Progress Reports

Reporting Period: 
Operational Milestone #1

Grant Application Details

Application Title: 
  • Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients
Public Abstract: 

Therapeutic Candidate or Device

HSC gene modified by a lentiviral vector which encodes a triple combination of HIV-resistance genes and a pre-selective marker.

Indication

HIV in AIDS-lymphoma patients.

Therapeutic Mechanism

Bone marrow transplants are standard of care for AIDS-lymphoma patients providing a cure for the patient's lymphoma. A purified population of triple combination anti-HIV lentiviral vector transduced CD34+ HSCs will safely engraft, divide, and differentiate in vivo into a pool of mature myeloid and lymphoid cells. This newly generated HIV-resistant immune system will be capable of blocking further HIV infection.

Unmet Medical Need

HIV continues to be a public health problem worldwide with no effective vaccine or cure available. Despite anti-retroviral therapy prolonging lives of patients, it is not curative. HIV stem cell gene therapy provides the potential to replace a patients immune system with one resistant to HIV.

Project Objective

To complete a Phase I clinical trial.

Major Proposed Activities

  • Evaluate the coorelatives of transplanted cells including DNA, immune, and virological monitoring.
  • To conduct a Phase I study of safety, feasability, and efficacy of our product in AIDS-lymphoma patients.
  • To manufacture and quality control GMP grade anti-HIV lentivector and clinical grade HIV-resistant HSC.
Statement of Benefit to California: 

In California, the number of HIV infected individuals continues to increase. As anti-retroviral drugs are not curative, these individuals still have to deal with the emotional, financial, and medical consequences. Our HIV stem cell gene therapy approach comprises the transplantation of a purified population of HIV-resistant blood forming stem cells which would generate an HIV-resistant immune system in a patient’s body. This would be significantly compelling to the state of California.