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CMP-CPS-001
- Development stage
- Phase 1
- Lead developer
- CAMP4 Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
CMP-CPS-001 is an antisense oligonucleotide (ASO) therapeutic candidate developed by CAMP4 Therapeutics for the treatment of urea cycle disorders (UCDs). It is designed to amplify CPS1 mRNA by targeting regulatory RNAs, thereby increasing the expression of carbamoyl phosphate synthetase 1 (CPS1), a key enzyme that catalyzes the first step in the urea cycle. By upregulating CPS1, CMP-CPS-001 aims to improve or restore urea cycle activity and lower toxic ammonia levels in patients with UCDs. The drug leverages CAMP4’s proprietary RAP Platform for discovery and design. CMP-CPS-001 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA and is currently being evaluated in a Phase 1 clinical trial in healthy volunteers[1][2][3][4][6][7].
- Other names
- CPS1-ASOCPS-1-ASOCPS 1-ASO
02
Targets
CPS1-IT1 (Carbamoyl-phosphate synthetase 1 intronic transcript 1)
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