Spirovant Sciences has announced the commencement of its SAAVe Phase I/II study, with the first patient receiving a dose of SP-101 in combination with an augmenter to treat cystic fibrosis.
An inhalable adeno-associated virus (AAV) gene therapy, SP-101 has been engineered for its tropism in human airway epithelia.
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The augmenter, doxorubicin is set to promote high expression of the functional cystic fibrosis transmembrane conductance regulator (CFTR) transgene.
The multicentre, open-label study involves dose escalation and expansion to assess the safety and tolerability of SP-101 plus the augmenter in cystic fibrosis patients ineligible for CFTR modulator therapy or for whom such treatments are inadequate.
Secondary endpoints of the trial include measurements of lung function, quality of life, and bronchoscopy biomarkers.
Spirovant Sciences chief scientific officer Roland Kolbeck said: “The dosing of the first patient in the SAAVe Phase I/II clinical trial is a major milestone for Spirovant and for the cystic fibrosis patient community.
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By GlobalData“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.”
The company’s ‘AAV + Augmenter’ platform helps develop customised therapies for cystic fibrosis and other respiratory conditions. The combination is designed to achieve high gene expression and enhance the effectiveness of the treatment across various respiratory conditions.
SP-101 has been optimised to address the challenges previously hindering the clinical viability of gene therapy for cystic fibrosis, aiming to treat its root cause across a broad spectrum of CFTR mutations.
Cystic fibrosis is a significant genetic disorder that shortens life expectancy and affects more than 100,000 individuals globally. This progressive multi-organ condition primarily impacts the lungs, digestive system, and reproductive tract.
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