Target intelligence / Profile preview

Optic Atrophy 1 Protein

Molecular classification
GTPase, Dynamin-related protein
01

Overview

The OPA1 protein is a dynamin-related GTPase localized to the inner mitochondrial membrane. It is essential for maintaining normal mitochondrial morphology and function. OPA1 mediates the fusion of mitochondrial inner membranes and regulates cristae structure, which is crucial for efficient oxidative phosphorylation. It also helps maintain mitochondrial DNA integrity and participates in controlled cell death (apoptosis). Mutations in OPA1 cause autosomal dominant optic atrophy (DOA) and have been implicated in neurodegenerative diseases. Therapeutic approaches under investigation include antisense oligonucleotides designed to increase functional OPA1 protein levels for DOA treatment.

Other names
Dynamin-like 120 kDa proteinOptic atrophy 1Mitochondrial dynamin-like GTPase
02

Mechanism of action

Antisense oligonucleotides designed to increase functional OPA1 protein levels.

03

Biological functions

Mitochondrial inner membrane fusionCristae remodelingApoptosis regulationMaintenance of mitochondrial DNA integrityRegulation of oxidative phosphorylation
04

Disease associations

Autosomal dominant optic atrophy (DOA)Neurodegenerative diseaseParkinson's diseaseSensorineural hearing lossAtaxiaNeuropathyOphthalmoplegiaMyopathy
05

Safety considerations

Mitochondrial dysfunction
06

Interacting drugs

STK-002 (antisense oligonucleotide)

Beyond the preview

Go deeper on Optic Atrophy 1 Protein.

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 Optic Atrophy 1 Protein.

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