Positive and negative controls for ssASOs and Gapmers that can be used for in vitro tox studies
Click below for download: Please feel free to use the ASOs on this list for the toxicity studies in your lab. If you have any ASOs that...
top of page
Collaborative Resources
Illustrated Examples to Download
Mar 19
N1C Preclinical Workgroup: Preclinical Modeling Systems
Click below for download
Mar 19
N1C Preclinical Workgroup: Ordering ASO Checklist
Click below for download
Mar 19
Mar 13
Community Job Openings
Welcome to our community job board where we curate relevant job openings to share with our community. If you have a job post to share, we...
Dec 20, 2023
N1C Patient Identification Workgroup: Compilation of Useful Tools
Click below for download: Disclaimer: This sample document is the work product of the N=1 Collaborative. This document is intended to...
Nov 28, 2023
Community Events
Updated November 28, 2023 FDA Clinical Investigator Training Course (CITC) 2023: December 6-7 2023 ABOUT THIS EVENT This course is...
Oct 4, 2023
‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
In 2014, the National Institutes of Health posted an estimate that at the current pace of drug development and approval, it would take...
Oct 4, 2023
The Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study
This study uses real world data to examine healthcare utilization relative to timing of diagnosis in seven RDs based on anticipated...
Oct 4, 2023
All of Us and the Promise of Precision Medicine:
Achieving Equitable Access for Federally Qualified Health Center Patients Click below for download: Disclaimer: This document is the work...
Oct 4, 2023
MAKING GENETIC THERAPIES AFFORDABLE AND ACCESSIBLE
The IGI brings together scientists and innovators from diverse disciplines to unlock the potential of CRISPR technology to solve some of...
Feb 10, 2023
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs
Antisense oligonucleotides (ASOs) can modulate pre-mRNA splicing. This offers therapeutic opportunities for numerous genetic diseases,...
Dec 15, 2022
Illustrated Example for n=1 Clinical Protocol
Click below for download: Disclaimer: This document is the work product of the N=1 Collaborative (the “N1C”), and is offered as an...
Dec 15, 2022
Illustrated Example for n=1 Consent Form
Click below for download: Disclaimer: This document is the work product of the N=1 Collaborative (the “N1C”), and is offered as an...
Jul 13, 2022
Clinical Recommendations Draft Guidance
IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases:...
Dec 6, 2021
CMC Recommendations Draft Guidance
Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating...
Apr 25, 2021
Nonclinical Testing Draft Guidance
Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases...
bottom of page