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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 by Korro Bio, Inc. for inborn urea cycle disorders, a group of rare inherited conditions. It is currently in Phase 1 clinical development and has not been approved by the FDA.
KRRO-121 is being developed for inborn urea cycle disorders, including ornithine transcarbamylase deficiency, arginase 1 deficiency, NAGS deficiency, carbamylphosphate synthetase I deficiency, citrin deficiency, and other related enzyme and transporter defects that impair the urea cycle.
KRRO-121 targets GLUL, the gene encoding the enzyme glutamine synthetase. By acting on this target, the small molecule is designed to address the impaired ammonia processing that characterizes inborn urea cycle disorders.
KRRO-121 is being developed by Korro Bio, Inc., a biopharmaceutical company that trades on the Nasdaq under the ticker symbol KRRO. The company is the sponsor of the clinical program evaluating the drug in urea cycle disorders.
KRRO-121 is in Phase 1 development. It is an investigational drug and has not received FDA approval. The ongoing study is a Phase 1/2 trial, and no trials of KRRO-121 have been completed to date.
KRRO-121 is being evaluated in a single recruiting Phase 1/2 study, NCT07773246, titled a Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD. The trial is placebo-controlled, randomized, double-blind, and plans to enroll 30 participants in Australia.