Precision BioSciences
DTILPhase 2Precision BioSciences leverages its novel ARCUS genome editing platform to create precise gene insertion, knockout, and repair therapies. The company has achieved significant clinical milestones, including FDA Fast Track and Orphan Drug designations for its lead programs, and has demonstrated early clinical safety and antiviral activity for PBGENE-HBV. Its strategy focuses on advancing its wholly-owned pipeline in genetic diseases while maintaining partnerships, such as with TG Therapeutics, to validate its platform in other therapeutic areas.
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AI Company Overview
Precision BioSciences leverages its novel ARCUS genome editing platform to create precise gene insertion, knockout, and repair therapies. The company has achieved significant clinical milestones, including FDA Fast Track and Orphan Drug designations for its lead programs, and has demonstrated early clinical safety and antiviral activity for PBGENE-HBV. Its strategy focuses on advancing its wholly-owned pipeline in genetic diseases while maintaining partnerships, such as with TG Therapeutics, to validate its platform in other therapeutic areas.
Technology Platform
ARCUS is a proprietary genome editing platform using engineered homing endonucleases to precisely insert, remove, or repair DNA in living cells and tissues, noted for its small size and high specificity.
Pipeline
5| Drug | Indication | Stage | Watch |
|---|---|---|---|
| PBGENE-DMD (IV) | Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD | Phase 1/2 | |
| Fludarabine + Cyclophosphamide | Non-Hodgkin's Lymphoma, Relapsed | Phase 1/2 | |
| PBGENE-HBV | HEPATITIS B CHRONIC | Phase 1 | |
| Fludarabine + Cyclophosphamide + Nirogacestat | Relapsed/Refractory Multiple Myeloma | Phase 1 | |
| PBGENE-HBV | Chronic HBV Infection | Pre-clinical |
Funding History
4Opportunities
Risk Factors
Competitive Landscape
Precision faces competition from large biopharma and other gene editing specialists. In HBV, competitors include Arrowhead/Janssen (RNAi) and Intellia (gene editing). In DMD, competitors include Sarepta (gene therapy) and CRISPR/Editas (gene editing). Differentiation is based on ARCUS's specific nuclease properties and its in vivo approach targeting muscle stem cells for durability.