Target intelligence / Profile preview

Carbonic anhydrase 5A, mitochondrial (None)

Target
None
Molecular classification
Enzyme, Metalloprotein
01

Overview

Carbonic anhydrase 5A, mitochondrial (CA5A) is a zinc metalloenzyme that catalyzes the reversible hydration of carbon dioxide to bicarbonate within mitochondria. It plays a crucial role in providing bicarbonate for carbamoyl phosphate synthetase I (urea cycle) and pyruvate carboxylase (gluconeogenesis), among other enzymes. CA5A is primarily expressed in the liver, and its deficiency leads to hyperammonaemia due to impaired ammonia detoxification. Treatment includes carglumic acid administration to support the urea cycle.

Other names
CA5CA5ADCAVCAVAGS1-21A4.1carbonic anhydrase VA/VA-mitochondrial
02

Mechanism of action

Carglumic acid enhances the activity of carbamoyl phosphate synthetase I, bypassing the need for CA5A-produced bicarbonate in the urea cycle and thus reducing hyperammonaemia.

03

Biological functions

CatalysisCarbon dioxide hydrationBicarbonate productionUreagenesis supportGluconeogenesis supportAcid-base balance
04

Disease associations

Carbonic anhydrase VA deficiencyHyperammonaemia
05

Safety considerations

Potential for hyperammonaemia recurrence despite treatmentVaried efficacy across patients due to residual enzyme activityNeed for careful monitoring to prevent metabolic decompensation
06

Interacting drugs

Carglumic acid

Beyond the preview

Go deeper on Carbonic anhydrase 5A, mitochondrial (None).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Carbonic anhydrase 5A, mitochondrial (None).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call