Characterization of RNA Therapeutics: Regulatory, Analytical, and Development Insights
RNA-based therapies—including siRNA, antisense oligonucleotides (ASOs), and aptamers—are transforming the treatment landscape across rare diseases, oncology, and metabolic disorders. With multiple FDA and EMA approvals already in place, the need for robust analytical and regulatory strategies is more critical than ever.
This scientific report highlights opportunities and current practices in RNA therapeutic characterization, guiding developers, CROs, and analytical service providers to align with regulatory expectations. Drawing on FDA submission case studies and established compendial methods, the report outlines key considerations for successful drug development.
Key Insights from the Report:
- Regulatory Landscape: Summaries of FDA and EMA decision documents for siRNA, ASO, and aptamer products provide a framework for submissions.
- Analytical Workflows
- Identity: SE-HPLC, LC–MS, Tm analysis for strand confirmation
- Purity & Impurities: HPLC–MS methods to detect truncations & modifications
- Stability: Stress testing under thermal, oxidative & freeze-thaw conditions
- Safety: Endotoxin, sterility & bioburden assays
- Higher-Order Structures: NMR, DLS & FIA for structure/aggregate assessment
- Protein Metrics Software: Protein Metrics Byos Oligo workflow, purpose-built for nucleic acid characterization. By integrating Protein Metrics Software into analytical workflows, developers gain automation, reproducibility, and confidence in meeting regulatory expectations
ACRNS is an Authorized distributor of Protein Metrics Software in India. For more information, email us at info@acrnstech.com or sales@acrnstech.com