SP-101, A Novel Adeno-Associated Virus Gene Therapy for the Treatment of Cystic Fibrosis, Mediates Functional Correction of Primary Human Airway Epithelia From Donors with Cystic Fibrosis

Inhalation of SP-101 Followed by Inhaled Doxorubicin Results in Robust and Durable hCFTRDR Transgene Expression in the Airways of Wild-Type and Cystic Fibrosis Ferrets

Repeat Dosing of AAV2.5T to Ferret Lungs Elicits an Antibody Response That Diminishes Transduction in an Age-Dependent Manner

Immunogenicity In Ferrets After Inhalation Delivery of SP-101

Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell

Inhalation of SP-101 followed by doxorubicin results in wide-spread SP-101 distribution and hCFTRΔR transgene expression in the airways of CF and non-CF ferrets

SP-101 gene therapy restores CFTR function in human CF airway epithelial cultures and drives hCFTRΔR transgene expression in the airways of CF and non-CF ferrets

Immunosuppression reduces rAAV2.5T neutralizing antibodies that limit efficacy following repeat dosing to ferret lungs

Restoring Ciliary Function: Gene Therapeutics for Primary Ciliary Dyskinesia

Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRDR gene delivery to adult ferret lungs
