Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases

On 26 April 2021 the FDA published draft guidance for sponsor-investigators.

The purpose of this guidance is to describe the nonclinical information that FDA recommends supporting an investigational new drug application (IND) for an antisense oligonucleotide being developed to treat a severely debilitating or life-threatening (SDLT) disease caused by a unique genetic variant where only a small number of individuals are prospectively identified (usually one or two). The investigational antisense oligonucleotide should be from a well-characterized chemical class for which there is substantial nonclinical information and clinical experience that is publicly available or to which the sponsor-investigator has a right of reference.

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