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FDA Guidance for Sponsor-Investigators: IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations



The purpose of this guidance is to provide recommendations regarding the chemistry, manufacturing, and controls (CMC) information that should be provided in an investigational new drug application (IND) submitted by a sponsor-investigator (hereafter referred to as sponsor) developing an individualized antisense oligonucleotide (ASO) drug product for a severely debilitating or life-threatening (SDLT) disease caused by a unique genetic variant where only a small number of individuals are prospectively identified (typically one or two)

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