Spirovant Sciences Begins Dosing in Phase 1/2 Trial for Aerosol Genetic Therapy for Cystic Fibrosis

A groundbreaking step in the treatment of cystic fibrosis has been achieved with the dosing of the first patient in Spirovant Sciences’ SAAVe Phase 1/2 clinical trial. This trial is evaluating SP-101, a novel gene therapy administered through inhalation, in combination with doxorubicin, to enhance the expression of the therapeutic gene in the lungs. This approach holds promise for patients who have not benefited from current treatment options.

Spirovant Sciences, based in Philadelphia, focuses on developing gene therapies for hereditary respiratory diseases. The SP-101 gene therapy is designed to treat cystic fibrosis by targeting the root cause of the disease. This therapy utilizes a recombinant adeno-associated virus (AAV) vector, chosen for its efficacy in human airway cells. The inclusion of doxorubicin as an augmenter helps in achieving high levels of expression of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is crucial for the potential clinical benefits of the therapy.

Chief Scientific Officer Roland Kolbeck emphasized the significance of this milestone, noting that SP-101's mutation-agnostic nature allows it to address a broad spectrum of cystic fibrosis mutations. This could provide a viable treatment option for approximately 10% of cystic fibrosis patients who do not respond to current CFTR modulators. These modulators, although effective for many, leave a subset of patients without any beneficial treatment.

SP-101 aims to overcome the historical limitations of gene therapy in treating cystic fibrosis. It targets the fundamental cause of lung disease in cystic fibrosis patients, offering hope for improved quality of life and better clinical outcomes. Associate Professor Claire Keating of Columbia University highlighted the trial's importance, acknowledging the contributions of the patients involved in the research. She expressed optimism that this innovative therapy could pave the way for effective treatments for those who currently lack adequate options.

The SAAVe Phase 1/2 trial is a multicenter, open-label study involving dose escalation and expansion to determine the safety and tolerability of SP-101 combined with doxorubicin. Secondary objectives include evaluating lung function, quality of life, and bronchoscopy biomarkers in patients who are not eligible for CFTR modulator therapy or for whom these modulators are inadequate.

Spirovant's AAV + Augmenter platform underpins the development of SP-101. This platform is tailored to enhance gene expression and improve the therapeutic effectiveness of treatments for cystic fibrosis and other respiratory conditions. The company is leveraging this technology to create customized therapies that address the unique challenges of these diseases.

Cystic fibrosis is a severe genetic disorder affecting over 100,000 people globally. Characterized by mutations in the CFTR gene, it leads to the production of thick mucus that impairs lung function, causes persistent infections, and results in progressive lung damage. While CFTR modulators have brought relief to many patients, they are not universally effective, leaving a significant unmet need for new treatments. The introduction of SP-101 could represent a major advancement for those who remain without effective therapy.

Spirovant Sciences continues to push the boundaries of gene therapy for respiratory diseases. With SP-101, the company is addressing the limitations of past gene therapies and striving to offer new hope for cystic fibrosis patients. Their innovative approach exemplifies the potential of gene therapy to transform the treatment landscape for hereditary respiratory diseases.