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Caribou-sponsored investigational therapy
This investigational therapy targets the CRISPR-Cas12 system to modulate gene expression.
This investigational therapy targets the CRISPR-Cas12 system to modulate gene expression. Used for Treatment of sickle cell disease.
At a glance
| Generic name | Caribou-sponsored investigational therapy |
|---|---|
| Sponsor | Caribou Biosciences, Inc. |
| Target | CRISPR-Cas12 system |
| Modality | Biologic |
| Therapeutic area | Genetics, Gene Therapy |
| Phase | Phase 2 |
Mechanism of action
The therapy utilizes a guide RNA to locate and cleave specific DNA sequences, allowing for precise gene editing. This approach has the potential to treat a wide range of diseases by correcting genetic mutations or silencing disease-causing genes.
Approved indications
- Treatment of sickle cell disease
Common side effects
- Off-target effects
- Immune response
Key clinical trials
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
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