eClinical Technology and Industy News

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

Excerpt from the Press Release:

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, an investigational novel recombinant adeno-associated virus (AAV) gene therapy selected for its tropism to human airway epithelia, at the 2021 North American Cystic Fibrosis Conference (NACFC 2021). In oral and poster presentations, Spirovant summarized recent pre-clinical data suggesting SP-101 functionally corrects human CF airway epithelia. In addition, the presentations showed that CFTR expression and CF correction exhibited by SP-101 are dose responsive and durable, demonstrating that SP-101 is a promising drug development candidate. SP-101 is intended for inhalation in patients with CF who do not benefit from treatment with small molecule CFTR modulators, which is approximately 20% of all patients with CF. A copy of the presentation slides and poster are available on the Spirovant website.

“These exciting data are the first preclinical models showing relevant levels of CFTR expression,” said Roland Kolbeck, PhD, Chief Scientific Officer of Spirovant Sciences. “With this information in hand, we believe that SP-101 holds great promise for the many patients living with CF, particularly those who have alleles with nonsense – or Class I – mutations or otherwise do not respond well to existing CFTR modulators.  As we continue to expand our scientific team, we plan to advance the development of this promising treatment and future programs for inherited respiratory disease.”

About Spirovant Sciences, Inc.
Spirovant is a gene therapy company focused on changing the course of cystic fibrosis and other respiratory diseases. The company’s current investigational gene therapy technologies are designed to overcome the historical barriers that have prevented effective genetic treatments for cystic fibrosis. Spirovant’s lead programs are in development for cystic fibrosis. Spirovant is a wholly-owned subsidiary of Sumitovant Biopharma Ltd., which is itself a wholly-owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Spirovant is located in Philadelphia, PA. More information is available at

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