WP 1:
• Created cGMP-compliant culture and differentiation media with 12-18 month shelf-life
• Achieved scalable iPSC expansion and cardiomyocyte production from 20mL to 500mL cultures
• Established robust residual iPSC assay, developed potency assay
• Created GMP-compliant SOPs, developed 24-hour cold transport protocols
• Successfully achieved cryopreservation
WP2:
• established standardized quality control protocols
• Adapted co-culture protocols to assess T cell alloresponses
• enabled standardized immune profiling protocols across multiple research sites, and created reference datasets for AI-based immune prediction algorithms
• Provided essential immune data for developing predictive models and advancing clinical translation of HLA-homozygous iPSC-CM therapies with robust immune characterization and standardized protocols
WP3:
• developed comprehensive mutation categorization system for therapeutic safety
• Successfully created HLA-homozygous iPSC suicide lines
• Developed scalable GMP-compliant gene, and validated production protocols for iPSC-derived cardiomyocyte aggregates
• Completed tumorigenicity studies in NSG mice and pig models, demonstrated no life-threatening arrhythmias in animal studies, and established comprehensive quality control for clinical-grade cell products
WP4:
• Established Target Product Profile (TPP), and created structured documentation system for IMPD development
• Completed preliminary documentation for PEI Scientific Advice request
• Developed SOPs for toxicity and carcinogenicity studies
• Established regulatory-ready documentation for clinical trial application
WP5:
• Conducted comprehensive FTO analysis, and established ongoing IP monitoring and strategic guidance
• Optimized GMP-compliant iPSC banking process to produce banks of up to 360 cryovials
• Created cost-effectiveness analysis framework for therapeutic development
WP6:
• Organized General Assembly meetings and quarterly consortium meetings, and maintained transparent communication with EU authorities
• Managed project website with multimedia content, organized HEAL symposium at GSCN 2024 conference, produced 24 scientific publications and multiple conference presentations, and maintained active social media presence across LinkedIn, X, and YouTube
• Implemented comprehensive risk monitoring and mitigation framework
• Maintained comprehensive project documentation on SharePoint, and ensured timely regulatory submissions and progress reports
