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RBG-005
- Development stage
- Preclinical
- Lead developer
- RophiBio
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
RBG-005 is a preclinical-stage gene therapy candidate developed by RophiBio for the treatment of Sjögren's syndrome. The therapeutic approach utilizes an adeno-associated virus serotype 2 (AAV-2) vector to deliver small interfering RNA (siRNA) or RNA interference (RNAi) components designed to silence or reduce the expression of Bone Morphogenetic Protein 6 (BMP-6). Overexpression of BMP-6 in the salivary glands is associated with the pathogenesis of Sjögren's syndrome, leading to glandular dysfunction and dryness. By targeting BMP-6, RBG-005 aims to restore salivary and lacrimal gland function.
- Other names
- BMP-6-targeting gene therapyBMP6-targeting gene therapyBMP 6-targeting gene therapy
02
Targets
BMP6 (Bone morphogenetic protein 6)
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