Protein / target
Carbamoyl-phosphate synthase [ammonia], mitochondrial
Protein at a glance
Biological role
Carbamoyl-phosphate synthase (glutamine-hydrolyzing)
Strongest disease association
Venous Thromboembolism
Therapeutic position
Established drug target
Derived from structured UniProt, Open Targets and literature data on this page.
Protein profile
Canonical identity and biological annotation from UniProt.
Function overview
Involved in the urea cycle of ureotelic animals where the enzyme plays an important role in removing excess ammonia from the cell
Subcellular location
Domains and Gene Ontology detail (53)Hide
Domains & features
Gene Ontology
- Ccytoplasm
- Ccytosol
- Cmitochondrial inner membrane
- Cmitochondrial matrix
- Cmitochondrial nucleoid
- Cmitochondrion
- Cnucleolus
- Cplasma membrane
- Cprotein-containing complex
- FATP binding
- Fcalcium ion binding
- Fcarbamoyl-phosphate synthase (ammonia) activity
Biological roles
What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.
View supporting evidenceHide supporting evidence
Lipid & lipoprotein metabolism
- ·phospholipid binding
- ·triglyceride catabolic process
Growth-factor signalling
- ·cellular response to fibroblast growth factor stimulus
Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.
Approved medicines with mapped indications
Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.
Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.
Drugs targeting this protein
How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.
Carbamoyl-phosphate synthase [ammonia], mitochondrial positive allosteric modulator
Indicated for Amino Acid Metabolism, Inborn Errors
ChEMBL mechanism, action type, target identity and approved indications. Open Targets clinical status.
Translational evidence
Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.
Strongest disease associations · via encoding gene CPS1
Gene-level evidence surfaced through the gene CPS1that encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.
View evidence synthesis (5)Hide
The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the per-type scores above show the calculation.
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Drug development
1 compounds recorded · 1 approved
View all recorded compounds (1)Hide
Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.
Tractability
Small molecules — Strong
Antibodies — Emerging
Protein degraders — Emerging
View underlying tractability evidence (11)Hide
Raw Open Targets tractability assessment buckets, by modality.
Clinical trials
Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.
ClinicalTrials.gov via the drug-target graph.
What's happening now
Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for a drug that targets this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.
- Regulatory approval
Approval: Ucedane (EMA)
- Regulatory approval
Approval: Carbaglu (EMA)
Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.