Spirovant Sciences is developing gene therapies for cystic fibrosis and other pulmonary diseases using a proprietary Adeno-Associated Virus (AAV) + Augmenter platform that enhances the expression of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in airway epithelia. The company addresses the limitations of existing genetic treatments by providing a targeted approach to restore CFTR function in patients who do not benefit from current therapies.
Funding
$4.5M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Existing treatments for cystic fibrosis (CF) and other pulmonary diseases have limitations in effectively restoring the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, particularly for patients who do not respond to current therapies. Traditional genetic treatments face challenges in achieving sufficient expression of the CFTR protein in airway epithelia.
Solution
Spirovant Sciences is developing gene therapies utilizing a proprietary Adeno-Associated Virus (AAV) + Augmenter platform to enhance CFTR protein expression in airway epithelia. The AAV + Augmenter platform consists of an optimized transgene payload, a novel AAV capsid, and an augmenter, collectively designed to drive high expression of the functional CFTR protein. This approach aims to overcome historical barriers that have prevented effective genetic treatments for cystic fibrosis, offering a targeted solution for patients who do not benefit from small molecule modulators. Spirovant's lead program is specifically designed to address the unmet needs in CF treatment by restoring CFTR function. The company's investigational, aerosol-delivered genetic medicine is currently in Phase 1/2 clinical trials.
Target Audience
The primary target audience includes patients with cystic fibrosis and other pulmonary diseases, particularly those who do not respond to existing small molecule modulator therapies, as well as the medical professionals who treat them.
Features
- Optimized transgene payload for enhanced CFTR protein expression.
- Novel AAV capsid designed for targeted delivery to airway epithelia.
- Augmenter component to further drive high levels of functional CFTR protein.
- Aerosol delivery method for direct administration to the lungs.
- Currently in Phase 1/2 clinical trials (SAAVe trial) for cystic fibrosis.
- Orphan Drug and Rare Pediatric Disease Designations granted by the FDA for SPIRO-2101.