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HGTV-43
- Development stage
- Unknown
- Lead developer
- Battery Ventures
- Modality
- Small Molecules, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
HGTV-43 is an investigational gene therapy developed by Enzo Therapeutics for the treatment of HIV-1 infection. It utilizes a proprietary vector (StealthVector HGTV43) to deliver anti-HIV-1 antisense RNA genes into autologous blood stem cells ex vivo. These engineered stem cells are then engrafted back into the patient’s bone marrow, where they produce progeny immune CD4 T cells containing the antisense RNA genes. The circulating engineered immune cells express anti-HIV-1 antisense RNA, which inhibits replication of HIV in these cells and confers resistance to infection. Preclinical studies demonstrated resistance to HIV in human immune cells with inserted antisense genes, and early-phase clinical trials showed successful engraftment and long-term persistence of modified immune cells in patients[1][2][3]. The drug is currently in Phase II clinical development for HIV-1 infections[3].
- Brand names
- StealthVector
- Other names
- StealthVector HGTV43
02
Targets
HIV-1 RNA (HIV-1 RNA genome)
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