A $6 million grant to Dr. Matthew Porteus and his team at Stanford University to develop an innovative stem cell-based gene correction therapy for chronic sinusitis in Cystic Fibrosis (CF) patients.  
 
CF is a common genetic disease that currently lacks a cure, causing patients to spend their lives managing symptoms. Despite significant advancements in CF therapies over the past two decades, effective treatments for serious sinus disease remain limited.

This new therapy aims to provide new treatment options for CF patients with specific mutations unresponsive to current treatments or those who cannot tolerate existing therapies.  

Although sinus disease in CF does not directly affect mortality, it significantly impacts quality of life and can have implications for lower respiratory tract health.  

The proposed approach aims to permanently correct CF transmembrane conductance regulator (CFTR) mutations, offering potentially transformative improvements over current treatments with longer-lasting effects. 

“The California Institute for Regenerative Medicine (CIRM) is delighted to support this pioneering gene-corrected sinus airway-based stem cell therapy for chronic sinusitis in CF patients. This innovative approach holds the potential to improve CF patients’ quality of life beyond current treatments,” said Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM. 

“We are thrilled to have assembled an outstanding multi-disciplinary team through the Stanford Center for Definitive and Curative Medicine (CDCM) to bring this stem-cell-based product to people with cystic fibrosis. Partnering with CIRM to address this large unmet medical need has been highly synergistic,” said Dr. Porteus.