GBT601 + PT2399

1 paper3 findings

C, W, M, H: cells, worms or flies, mice, humans. Dark: independent labs found the same change there. Light: one lab or one study. Outline: nothing yet.

Labs are independent when their papers share no last author and no institution.

Against: findings that point the other way from most findings on the same outcome.

No results posted: trials that ended more than a year ago with no results on ClinicalTrials.gov and no paper in ARN’s record.

Company ties: findings with an author at a company, or from a paper whose competing-interests statement declares ties to one.

1 lab in 1 country

Animals1

Outcomes

Motor phenotypes

upin mice1

1 study

GBT601 + PT2399 improves motor phenotypes in mice.
Animalsin a mouse model of Parkinson's disease due to α-synuclein toxicity
Oxygen-limiting drug combination extends lifespan in mice
Proceedings of the National Academy of Sciences of the United States of America9 Oct 2026

Lipid peroxidation

downin mice1

1 study

GBT601 + PT2399 decreases lipid peroxidation in mice.
Animalsin a mouse model of Parkinson's disease due to α-synuclein toxicitybrain
Oxygen-limiting drug combination extends lifespan in mice
Proceedings of the National Academy of Sciences of the United States of America9 Oct 2026

Latest

1 in 12 months

PNAS5 h

Oxygen-limiting drug combination extends lifespan in mice

In mice with severe mitochondrial disease, a two-drug treatment extended median lifespan from 62 days to 158 days.In mice with severe mitochondrial disease, a two-drug treatment extended median lifespan from 62 days to 158 days.

Experiments in miceInterventions

Paper

GBT601 + PT2399

Outcomes

Latest

Oxygen-limiting drug combination extends lifespan in mice

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