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

Poster: 621: Administration of SP-101 and doxorubicin results in robust and durable human cystic fibrosis transmembrane conductance regulator minigene transgene expression in the airways of ferrets and corrects human cystic fibrosis airway epithelia in vitro. North American Cystic Fibrosis Conference 2022

Poster: 672: SP-101 activity increases in a dose dependent manner to both vector and doxorubicin dose. North American Cystic Fibrosis Conference 2022

Spirovant’s SP-101 pre-clinical data demonstrates great promise for individuals with cystic fibrosis (CF) who do not benefit from small molecule modulators

November 3, 2022 New pre-clinical data for SP-101, an investigational novel recombinant adeno-associated virus (AAV) gene therapy selected for its tropism to human airway epithelia for the treatment of people living with cystic fibrosis (CF), suggest co-administration of SP-101 with doxorubicin functionally corrects human CF airway epithelia by restoring chloride conductance to similar levels as […]
Abstract: 672: SP-101 activity increases in a dose dependent manner to both vector and doxorubicin dose. North American Cystic Fibrosis Conference 2022

Abstract: 621: Administration of SP-101 and doxorubicin results in robust and durable human cystic fibrosis transmembrane conductance regulator minigene transgene expression in the airways of ferrets and corrects human cystic fibrosis airway epithelia in vitro. North American Cystic Fibrosis Conference 2022
