Illustrated ExamplesPositive and negative controls for ssASOs and Gapmers that can be used for in vitro tox studies
Illustrated ExamplesN1C Preclinical Workgroup: Valuable Tools for the Preclinical Stage of Developing Potential Therapeutics Click below for download
Illustrated ExamplesCommunity Job OpeningsWelcome 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...
Illustrated ExamplesCommunity EventsUpdated November 28, 2023 FDA Clinical Investigator Training Course (CITC) 2023: December 6-7 2023 ABOUT THIS EVENT This course is...
Illustrated Examples‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
Illustrated ExamplesConsensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs
Regulatory GuidancesClinical Recommendations Draft GuidanceIND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases:...
Regulatory GuidancesCMC Recommendations Draft GuidanceInvestigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating...
Illustrated ExamplesNonclinical Testing Draft GuidanceNonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases...
Regulatory GuidancesAdministrative and Procedural Draft GuidanceIND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for...
