RNA Therapeutics Institute RNA Therapeutics From Concept to Clinic 2024

A translational, bench-to-bedside program covering the full lifecycle of RNA medicines. Faculty connect platform science (siRNA, ASO, mRNA, saRNA) with delivery, CMC, regulatory strategy, and clinical development to help teams move candidates efficiently and safely toward approval.
What You Will Learn

Modality selection and design: siRNA vs. ASO vs. (sa)mRNA—chemistries, modifications, and stability

Delivery systems: LNPs, GALNAc conjugates, polymers/peptides, local vs. systemic administration

Preclinical translation: in vitro/in vivo models, PK/PD, biodistribution, immunogenicity and off-target risk

CMC fundamentals: process development, analytics, release/stability testing, scale-up and tech transfer

Clinical development: FIH design, dose selection, biomarkers, patient selection, endpoints, and safety monitoring

Regulatory & quality: IND/IMPD packages, comparability, potency assays, pharm tox expectations, and risk management

Manufacturing operations: raw materials (lipids, nucleotides, enzymes), single-use workflows, contamination control

Data & tools: sequence optimization, secondary structure prediction, bioinformatics/AI for target and off-target assessment

Access & value: manufacturing cost drivers, IP strategy, partnerships, and commercialization pathways

Event Details

Format: Case-based lectures, panel Q&A, and short technical workshops

Structure: Concept/design → delivery/PKPD → CMC/manufacturing → clinical/regulatory → commercialization

Takeaways: Checklists, study-design templates, and CMC/quality worksheets for immediate use

Who Should AttendR&D scientists, CMC/quality leaders, translational and clinical teams, regulatory/strategy professionals, manufacturing/operations managers, platform and biotech founders, investors, and trainees entering the RNA space.
Why Attend

Replace trial-and-error with validated design and development playbooks

Anticipate regulatory and manufacturing pitfalls early to de-risk timelines

Bring back practical tools to align discovery, CMC, and clinical teams around the same plan

 
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