RNA Therapeutics Institute RNA Therapeutics From Concept to Clinic 2025

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 AttendR&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

* Detail: Schedule