Clinical expert panel on monitoring potential lung toxicity of inhaled oligonucleotides: consensus points and recommendations.

摘要: Oligonucleotides (ONs) are an emerging class of drugs being developed for the treatment a wide variety diseases including respiratory by inhalation route. As class, their toxicity on human lungs has not been fully characterized, and predictive biomarkers have identified. To that end, identification sensitive methods can detect in humans before any long term and/or irreversible side effects occur would be helpful. In light public's greater interests, Inhalation Subcommittee Oligonucleotide Safety Working Group (OSWG) held expert panel discussions focusing potential inhaled ONs assessing strengths weaknesses different monitoring techniques use during clinical evaluation ON candidates. This white paper summarizes key captures panelists' perspectives recommendations which, we propose, could used as framework to guide both industry regulatory scientists future research characterize monitor short lung response ONs.