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
Novel approach represents new potential for patients who do not benefit from current treatment options
November 14, 2024
Spirovant Sciences, a clinical-stage gene therapy company developing treatments for inherited respiratory diseases, today announced that the first patient has been dosed in its SAAVe Phase 1/2 clinical trial of SP-101 + Augmenter for the treatment of cystic fibrosis.
Delivered via inhalation, SP-101 is an investigational novel recombinant adeno-associated virus (AAV) gene therapy selected for its tropism, in human airway epithelia. The selected augmenter, doxorubicin, drives high expression of the functional cystic fibrosis transmembrane conductance regulator (CFTR) transgene to levels with a high potential of providing clinical benefit.
“The dosing of the first patient in the SAAVe Phase 1/2 clinical trial is a major milestone for Spirovant and for the cystic fibrosis patient community,” said Roland Kolbeck, PhD, Chief Scientific Officer of Spirovant Sciences. “This therapy is mutation agnostic and as such has the potential to treat a range of cystic fibrosis patients, including those who are not eligible for treatment with the CFTR modulators as well as those for whom the CFTR modulators are insufficient.”
Approximately 10% of individuals living with cystic fibrosis do not benefit from existing CFTR modulator therapies, leaving them without treatment options. SP-101 has been optimized to overcome challenges that have previously limited the clinical viability of gene therapy for cystic fibrosis, and has the potential to address the underlying cause of cystic fibrosis lung disease – thereby benefitting a broad range of patients with cystic fibrosis, independent of their specific CFTR mutations.
“Dosing our first patient with this unique new gene therapy treatment candidate is an exciting step forward for the cystic fibrosis community we support,” said Claire Keating, MD, Associate Professor of Medicine, and Co-Director, Gunnar Esiason Adult Cystic Fibrosis and Lung Center at Columbia University Irving Medical Center. “This successful dosing is a major development, advancing a novel potential treatment pathway that moves us towards our goal of effectively treating people with CF who currently are not eligible for other highly effective therapies. I am especially grateful to our patient, and all those participants who partner in this research, without whom the development of new CF medicines would not be possible,” added Dr. Keating.
The SAAVe Phase 1/2 clinical trial is a multicenter, open-label, dose escalation and dose expansion trial of SP-101 + Augmenter in patients with cystic fibrosis who are not eligible for CFTR modulator therapy or for whom the CFTR modulators are insufficient. The primary endpoint of the study is safety and tolerability. Secondary endpoints include lung function (ppFEV1), quality of life measurement (CFQ-R) and bronchoscopy biomarkers.
About Spirovant’s AAV Platform
The AAV + Augmenter platform provides Spirovant with the unique opportunity to design tailored therapies for cystic fibrosis and other respiratory diseases. The combination of the AAV with the augmenter is designed to drive high gene expression and enhance effectiveness of the treatment for a range of respiratory diseases.
About Cystic Fibrosis
Cystic fibrosis is a serious, life-shortening genetic disease affecting over 100,000 worldwide. It is a progressive multi-organ disease primarily impacting the lungs, digestive, and reproductive tract caused by loss-of-function mutations in the CFTR gene. The genetic mutations lead to a build-up of thick mucus that causes impaired lung function, persistent lung infections and repeated exacerbations. These complications result in progressive lung damage, hospitalizations, and ultimately respiratory failure. While recent therapies known as CFTR modulators have improved symptoms for many, they are not effective for all individuals with CF, leaving many without sufficient treatment options. For those who cannot benefit from these therapies, the disease burden remains high, with a pressing need for new and effective treatments
About Spirovant Sciences, Inc.
Spirovant is a gene therapy company developing treatments for respiratory diseases including cystic fibrosis. The company’s current investigational gene therapy technologies are designed to overcome the historical barriers that have prevented effective genetic treatments for cystic fibrosis and other respiratory diseases. Spirovant’s lead program is in development for cystic fibrosis using our proprietary AAV + Augmenter platform. More information is available at https://www.spirovant.com.
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