Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients
Grant Award Details
Grant Type:
Grant Number:
CLIN2-08289
Investigator(s):
Disease Focus:
Human Stem Cell Use:
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
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.