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Cal-1
- Development stage
- Phase 2
- Lead developer
- CSL Behring
- Modality
- Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
Cal-1 is an experimental gene therapy developed for the treatment and prevention of HIV infection. It involves autologous transplantation of hematopoietic stem/progenitor cells (CD34+) and/or CD4+ T lymphocytes that have been genetically modified ex vivo using a lentiviral vector encoding two anti-HIV components (LVsh5/C46). The first component knocks down CCR5 expression to prevent HIV entry into host cells; the second expresses C46 peptide to inhibit viral fusion. This dual mechanism aims to render immune cells resistant to HIV infection. The therapy was initially developed by Calimmune and has been investigated in early-phase clinical trials for acquired immunodeficiency syndrome (AIDS) but development appears discontinued or on hold[4][5].
- Other names
- Autologous CD34+ Haematopoietic Stem/Progenitor Cells + CD4+ T Lymphocytes Transduced with LVsh5/C46 - CalimmuneCal-1-CalimmuneCal1-CalimmuneCal 1-Calimmune
02
Targets
gp41 (Human immunodeficiency virus type 1 glycoprotein 41)
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