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Spirovant Sciences Doses First Patient in the SAAVe Phase 1/2 Clinical Trial of Its Investigational, Aerosol-Delivered Genetic Medicine for the Treatment of Cystic Fibrosis

SP-101, an AAV vector-based gene therapy delivered via inhalation, is being investigated in combination with doxorubicin, an augmenter to enhance transgene expression in the lungs

Press Releases

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

Press Releases

Spirovant Launches State-of-the-Art Laboratory and Headquarters

Philadelphia, PA, May 17, 2022 – Spirovant Sciences, a gene therapy company developing treatments for inherited respiratory diseases, including cystic fibrosis (CF), announced today the launch of its new state-of-the-art research laboratory and corporate headquarters in Philadelphia.

Press Releases

Spirovant Presents Preclinical Data Showing SP-101 Restores CF Transmembrane Conductance Regulator Function at the 2021 North American Cystic Fibrosis Conference

Philadelphia, PA, November 4, 2021 – Spirovant Sciences, a gene therapy company developing treatments and cures for inherited respiratory diseases including cystic fibrosis (CF), today presented SP-101,

Press Releases

FDA Grants Spirovant Sciences Orphan Drug and Rare Pediatric Disease Designations for SPIRO-2101 for Treatment of Cystic Fibrosis

PHILADELPHIA, Sept. 24, 2020 /PRNewswire/ — Spirovant Sciences, a gene therapy company developing treatments and cures for genetic lung diseases including cystic fibrosis, today announced that

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PRESS RELEASES

Spirovant Sciences Doses First Patient in the SAAVe Phase 1/2 Clinical Trial of Its Investigational, Aerosol-Delivered Genetic Medicine for the Treatment of Cystic Fibrosis

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

Spirovant Launches State-of-the-Art Laboratory and Headquarters

Spirovant Presents Preclinical Data Showing SP-101 Restores CF Transmembrane Conductance Regulator Function at the 2021 North American Cystic Fibrosis Conference

FDA Grants Spirovant Sciences Orphan Drug and Rare Pediatric Disease Designations for SPIRO-2101 for Treatment of Cystic Fibrosis

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PUBLICATIONS

Poster: Intratracheal administration of AAV2.5T-SP183-fCFTRΔR in combination with doxorubicincorrects the mucociliary clearance defect in cystic fibrosis model ferrets

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

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

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

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

Katherine Excoffon: Delivery of SP-101 restores CFTR function in human CF airway epithelial cultures and drives hCFTRΔR transgene expression in the airways of ferrets. North American Cystic Fibrosis Conference 2021. – Poster

Katherine Excoffon: Delivery of SP-101 restores CFTR function in human CF airway epithelial cultures and drives hCFTRΔR transgene expression in the airways of ferrets. North American Cystic Fibrosis Conference 2021. – Video

Optimization of Recombinant Adeno-Associated Virus-Mediated Expression for Large Transgenes, Using a Synthetic Promoter and Tandem Array Enhancers

Directed evolution of adeno-associated virus to an infectious respiratory virus