Feb 10Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOsAntisense oligonucleotides (ASOs) can modulate pre-mRNA splicing. This offers therapeutic opportunities for numerous genetic diseases,...
Dec 15, 2022Illustrated Example for n=1 Clinical ProtocolClick below for download: Disclaimer: This document is the work product of the N=1 Collaborative (the “N1C”), and is offered as an...
Dec 15, 2022Illustrated Example for n=1 Consent FormClick below for download: Disclaimer: This document is the work product of the N=1 Collaborative (the “N1C”), and is offered as an...
Jul 13, 2022Clinical Recommendations Draft GuidanceIND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases:...
Dec 6, 2021CMC Recommendations Draft GuidanceInvestigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating...
Apr 25, 2021Nonclinical Testing Draft GuidanceNonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases...