Spirovant Sciences is a biotechnology company focused on developing innovative gene therapies for respiratory diseases, particularly cystic fibrosis (CF). Their primary technology is the proprietary Adeno-Associated Virus (AAV) + Augmenter platform, which aims to deliver a fully functional copy of the CFTR gene directly to the respiratory system. This platform is designed to overcome historical challenges associated with gene therapy for CF, such as low efficacy and delivery issues. The AAV component is a replication-defective virus that can infect both dividing and non-dividing cells, eliciting minimal immune response, making it a suitable candidate for gene therapy. The addition of small molecule augmenters enhances the translocation of the AAV genome into the cell nucleus, significantly increasing transgene expression levels.

Spirovant's lead candidate, SP-101, represents a novel inhaled AAV gene therapy that combines the AAV2.5T capsid with a human CFTR minigene and is co-administered with doxorubicin, a proteasome inhibitor that boosts the production of functional CFTR protein. This innovative approach is mutation-agnostic, meaning it can potentially benefit all CF patients, regardless of their specific CFTR mutations. The company has made significant strides in its clinical development pipeline, with SP-101 currently undergoing Phase 1/2 trials, and has received orphan drug and rare pediatric disease designations from regulatory authorities, highlighting its potential impact on treating this life-threatening condition.

United States

Acquired by Sumitomo Dainippon Pharma of Japan. Spirovant Sciences is a gene therapy company focused on changing the course of cystic fibrosis and other respiratory diseases. The company's current focus is on developing novel gene therapies for cystic fibrosis and other pulmonary diseases. Spirovant is located in Philadelphia, PA's thriving Life Sciences Corridor and has a diverse management team, researchers, and partners.

Spirovant Sciences

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