UMass Chan RNAtx RNA Therapeutics From Concept to Clinic 2024

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Description

Include: 3 videos + 1 pdf, size: 84.43 GB by RNA Therapeutics Institute & UMass Chan Medical School

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) 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 Attend
R&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

+ Topics:

* Detail:

Day 3- RNATx Program

UMass Chan RNAtx RNA Therapeutics From Concept to Clinic 2024

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