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Pip6a-Ctrl
- Development stage
- Discontinued
- Lead developer
- University of Oxford
- 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, Peptide-Drug Conjugates → Peptide Conjugates → Peptides
- Administration
- Intravenous
01
Overview
Pip6a-Ctrl is a research-grade control conjugate designed to evaluate the efficacy and specificity of peptide-conjugated antisense oligonucleotides (ASOs). It comprises the Pip6a cell-penetrating peptide (CPP)—an arginine-rich peptide with a hydrophobic core—covalently linked to a phosphorodiamidate morpholino oligomer (PMO) containing a reverse GAC7 sequence. In studies of Myotonic Dystrophy Type 1 (DM1), it serves as a negative control for Pip6a-PMO-CAG; while the latter targets toxic CUG expansions in the DMPK gene to restore splicing and muscle function, Pip6a-Ctrl lacks a complementary target and demonstrates no therapeutic activity, thereby confirming that the benefits of the active drug are sequence-dependent.
- Other names
- Pip6a-PMO-GAC7Pip-6a-PMO-GAC7Pip 6a-PMO-GAC7Pip6a-conjugated reverse GAC7 PMOPip-6a-conjugated reverse GAC7 PMOPip 6a-conjugated reverse GAC7 PMO
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