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,...
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Illustrated Examples to Download
- 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Clinical Recommendations Draft Guidance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CMC Recommendations Draft Guidance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Nonclinical Testing Draft Guidance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...
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