RNA Therapeutics Institute RNA Therapeutics From Concept to Clinic 2025

+ Include: 3 videos + 1 pdf, size: 83.51 GB

+ Target Audience: R&D scientists, CMC/quality leaders, translational and clinical teams, regulatory/strategy professionals, manufacturing/operations managers

+ Information:

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

What You Will Learn

• Modality selection & design: choosing siRNA vs. ASO vs. (sa)mRNA vs. circRNA; chemistries, modifications, stability

• Delivery systems: LNPs, GALNAc conjugates, polymer/peptide carriers, local vs. systemic administration

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

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

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

• Regulatory & quality: IND/IMPD strategy, comparability, potency assays, pharm-tox expectations, risk management

• Manufacturing operations: raw materials, supply chain, single-use workflows, contamination control, cost drivers

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

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 Attend
R&D scientists, CMC/quality leaders, translational and clinical teams, regulatory/strategic affairs, manufacturing/operations managers, platform 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

• Align discovery, CMC, and clinical groups around practical, decision-ready tools

+ Topics:

*Note: these are continuous video recordings during the conference, they include individual lectures mentioned in the Detail section below

• June 25, 2025.mp4
• June 26, 2025.mp4
• June 27, 2025.mp4