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KRRO-121 · 1 trial · 11 indications
General toxicity: Any Grade 3 or higher adverse event (AE) per Common Terminology Criteria for Adverse Events (CTCAE); Serious adverse events: Any serious adverse event (SAE) assessed as related or possibly related to KRRO-121.
Any adverse event (AE) per Common Terminology Criteria for Adverse Events (CTCAE),
| Arm | Type | Description |
|---|---|---|
| KRRO-121 | EXPERIMENTAL | KRRO-121 is a GalNAc-conjugated RNA-editing oligonucleotide that recruits endogenous hepatic ADAR to edit mRNA, producing a de novo glutamine synthetase (GS) protein resistant to feedback-driven proteasomal degradation. By stabilizing hepatic GS, KRRO-121 augments ammonia clearance capacity independently of the underlying urea cycle enzyme defect. |
| Placebo | PLACEBO_COMPARATOR | Placebo |
| Name | Type | Description |
|---|---|---|
| KRRO-121 | DRUG | KRRO-121, subcutaneous injection |
| Placebo | DRUG | Placebo, subcutaneous injection |
Inclusion Criteria: * Adult male or female participants, 18 to 65 years of age * Healthy as determined by medical history, physical examination, vital signs, 12-lead electrocardiogram (ECG), and screening laboratory assessments, in the opinion of the Investigator. * Female participant of childbeari...
KRRO-121 is an investigational small molecule being developed for the treatment of inborn urea cycle disorders, a group of rare genetic conditions that impair the body's ability to remove ammonia from the blood. It is being studied in patients with various urea cycle disorders, including ornithine transcarbamylase deficiency, arginase 1 deficiency, and citrin deficiency.
KRRO-121 is a small molecule designed to address inborn urea cycle disorders, which involve deficiencies in enzymes or transporters of the urea cycle. The specific molecular target of KRRO-121 has not been disclosed in the available information.
KRRO-121 is being developed by Korro Bio, Inc., a biopharmaceutical company listed on the stock exchange under the ticker symbol KRRO. The company is conducting clinical research on this investigational therapy for urea cycle disorders.
KRRO-121 is in Phase 1 clinical development. A Phase 1/2 study is planned to evaluate the drug in healthy volunteers and patients with urea cycle disorders, but the trial has not yet started recruiting participants.
KRRO-121 is being studied in a Phase 1/2 clinical trial with the identifier NCT07773246. This randomized, double-blind, placebo-controlled study plans to enroll 30 participants in Australia, including healthy volunteers and patients with urea cycle disorders.
KRRO-121 is the primary name for this investigational drug. No alternative names have been reported for this asset in the available information.