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messenger RNA (alpha-synuclein)
- Development stage
- Preclinical
- Lead developer
- Factor Bioscience
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies
01
Overview
messenger RNA (alpha-synuclein) is a preclinical mRNA-based in vivo gene editing candidate being developed by Factor Biosciences Therapeutics for the treatment of Parkinson's disease. The therapy utilizes Factor's proprietary mRNA technology to deliver gene-editing components directly to cells in vivo, specifically targeting the alpha-synuclein (SNCA) gene or its regulatory pathways. By reducing the production or accumulation of alpha-synuclein protein aggregates, which are central to the pathogenesis of Parkinson's and other synucleinopathies, the drug aims to modify the course of the disease rather than just managing symptoms.
- Other names
- mRNA (alpha-synuclein)alpha-synuclein mRNA
02
Targets
SNCA (Alpha-synuclein)Alpha-synuclein messenger RNA 5′ untranslated region iron-responsive element (SNCA 5′ UTR IRE)
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