ACT 2024 -- Cell and gene therapies (CGT) offer an attractive approach to treating monogenic pulmonary diseases. Standard nonclinical inhalation (INH) exposure systems require greater volumes of test article than other parenteral routes of administration because multiple animals are simultaneously dosed on a single exposure system with innate inefficiencies. Because clinical treatment with CGT modalities will likely only require single or infrequent dose administration, intratracheal (IT) administration offers an alternative route for pulmonary delivery; however, IT dosing requires anesthesia. Customized administration techniques for IT administration to cynomolgus monkey (NHP) and nose-only inhalation to mice were set up for delivery of an adeno-associated virus (AAV). Results from the mouse study were presented previously, while here the hypothesis tested is regarding achieved lung doses and test article usage.

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