Connecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting
Selecting the right candidate: Which disease-causing variants are eligible for N=1 ASOs?
Tailored RNA therapies for ultrarare CNS diseases & mutations: a criteria framework for patient ID
Fast Track to Final Drug Product: Understanding Costs, Challenges, and Timelines in Personalized Medicine Manufacturing
"Control of starting material quality for oligonucleotide therapeutics" and "To Synthesis and Beyond…Production of oligos for ‘N-of-1’ therapies"
CMC Recommendations Draft Guidance
Preclinical Development for Personalized Therapy Using Disease-Relevant Cell Models
The Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
Models for Sustainable Discovery
N1C Preclinical Workgroup: Preclinical Modeling Systems
N1C Preclinical Workgroup: Ordering ASO Checklist
A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides
Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing
The Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs
Nonclinical Testing Draft Guidance
OTS Rare Disease N-of-1+ Briefing Document, Jan 2020
Regulatory Pathway: Where We Are Today
Approach to Drafting Regulatory Submissions and Challenges of Institutional Implementation
Illustrated Example for n=1 Clinical Protocol
Illustrated Example for n=1 Consent Form
Administrative and Procedural Draft Guidance
