BrugaGene
Precision therapy for genetic cardiac disease.
Top 5 Finalist - Pharmaceutical Industrial Case Study
Problem
Brugada Syndrome arises from SCN5A ion-channel mutations. Existing treatments manage symptoms without addressing root causes. Precision genetic therapy requires cardiac-specific delivery.
Overview
BrugaGene is a conceptual therapeutic strategy combining CRISPR-Cas9 gene editing with cardiac-targeted lipid nanoparticle delivery to address SCN5A mutations in Brugada Syndrome.
Concept
A single-dose CRISPR-Cas9 strategy delivered via cardiac-targeted lipid nanoparticles to correct SCN5A mutations and restore normal cardiac ion-channel function.
Scope
CRISPR-Cas9 therapy design, SCN5A mutation targeting, lipid nanoparticle formulation, cardiac-targeting peptide modification, precision genetic therapy frameworks, and pharmaceutical biotechnology concepts.
Contribution
Led CRISPR strategy development and nanoparticle cardiac-targeting design as part of a multidisciplinary biotechnology case team.
Reflection
This concept highlighted how emerging CRISPR technology and precision delivery systems can address previously intractable genetic cardiac diseases.