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cobomarsen
- Development stage
- Discontinued
- Lead developer
- Viridian Therapeutics
- Modality
- 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
- Administration
- Intravenous, Subcutaneous, Intratumoral Injection (in Early Studies)
01
Overview
Cobomarsen is a locked nucleic acid (LNA)-based oligonucleotide inhibitor of microRNA miR-155, developed as an antineoplastic agent. It specifically targets and binds to miR-155, an oncogenic single-stranded non-coding RNA that is overexpressed in several blood cancers. By inhibiting miR-155, cobomarsen silences its activity and prevents the translation of tumor-promoting genes, leading to cancer cell apoptosis and inhibition of tumor growth. The LNA modification increases the stability and affinity for its target compared to unmodified oligonucleotides. Cobomarsen was primarily investigated for T-cell lymphomas including cutaneous T-cell lymphoma (CTCL), mycosis fungoides (MF), adult T-cell leukemia/lymphoma (ATLL), chronic lymphocytic leukemia (CLL), and diffuse large B-cell lymphoma (DLBCL). Despite initial promise and orphan drug designation by the FDA for T-cell lymphoma, clinical development has been discontinued following lackluster efficacy results in phase 2 trials[1][3][4][5][7][8].
- Brand names
- cobomarsen
- Other names
- cobomarsen
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