Disease Focus: Infectious Disease
Engineering Lifelong Cellular Immunity to HIV
Research Objective We aim to uncover a therapeutic approach to attempt to treat and potentially cure HIV infection using gene modified blood forming stem cells to enhance the immune response against HIV. Impact The study will allow a potentially curative treatment for HIV infection, which currently doesn’t exist. This will eliminate the need to administer […]
Immunization strategies to prevent Zika viral congenital eye and brain disease
Research Objective Our objective is to utilize human iPSC-derived neural and ocular cells to identify growth attenuated and non-pathogenic Zika virus vaccine candidates that can prevent congenital ZIKV disease. Impact Currently, there are no therapies or vaccines available against ZIKV for human use. The human iPSC technology provides a unique opportunity to test the growth […]
A treatment for Zika virus infection and neuroprotection efficacy
Research Objective We propose to determine the impact of the Zika virus during human neurodevelopment and to test a FDA-approved therapeutic candidate to treat Zika infection. Impact A drug to treat/cure Zika infection and for neuroprotection. Major Proposed Activities To determine the molecular and cellular alterations caused by the Zika virus in the human developing […]
Immunotherapy for HIV infection using engineered hematopoietic stem/progenitor cells
Research Objective The therapeutic candidate proposed here is hematopoietic stem/progenitor cells engineered to encode for HIV-specific T cell receptors. Impact The success of the proposed studies will test the efficacy of an approach to provide long-lasting functional cure for HIV infection, obviating the need for anti-retroviral therapy. Major Proposed Activities Test if engineered hematopoietic stem/progenitor […]
Hematopoietic Stem/Progenitor Cell-Based Chimeric Antigen Receptor Gene Therapy for HIV Infection
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 […]
Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients
Translational Candidate Cytomegalovirus antiviral FD-86: DNA Oligonucleotide Therapy Area of Impact HSCT transplant rejection and childhood cognitive and hearing impairment caused by Cytomegalovirus Mechanism of Action The candidate oligonucleotide therapy, FD-86 disrupts viral IE feedback circuitry and breaks homeostatic control of cytotoxic IE proteins inducing apoptosis only in infected cells. Unmet Medical Need Treatment-resistant CMV […]
62nd Midwinter Conference of Immunologists
Evaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV
Therapeutic Candidate or Device Cytomegalovirus (CMV)-specific T cells that express a chimeric antigen receptor (CAR) which targets and eliminates HIV-infected cells Indication Management of human immunodeficiency virus (HIV)/acquired immunodeficiency syndrome (AIDS) Therapeutic Mechanism Persons with HIV (PWH) lose immunity because of decreased T lymphocyte function. We propose to treat them with CAR T cells which […]
Phase 1 Clinical research program for functional cure of HIV with EBT-101, in-vivo gene therapy
Therapeutic Candidate or Device EBT-101 is a novel genome editing therapeutic targeting integrated HIV-1 genome to achieve sustained virologic reduction to enable functional cure. Indication EBT-101 is intended to deplete the reservoir of integrated HIV-1 proviral DNA in immune reconstituted virally suppressed HIV-1 positive individuals. Therapeutic Mechanism EBT-101 is an in vivo gene therapy product […]
Anti-HIV duoCAR-T cell therapy for HIV infection
Therapeutic Candidate or Device HIV-specific CAR-T cells Indication Management of HIV infection Therapeutic Mechanism We will modify T cells such that they are able to directly control HIV in the absence of therapy. Should this work, these cells will result in long-term control of HIV in absence of any ongoing treatment, a version of a […]