Cystic Fibrosis Foundation Invests $5 Million in Sionna Therapeutics to Develop New CFTR Modulator Therapy

New modulators in development by the CF-specific company could provide unique treatment options for the underlying cause of cystic fibrosis for many people with the disease

BETHESDA, Md.--(BUSINESS WIRE)--The Cystic Fibrosis Foundation today announced its investment of $5 million in Sionna Therapeutics, a company founded in 2019. The company is focused exclusively on developing cystic fibrosis transmembrane conductance regulator (CFTR) modulators, a type of therapy that treats the underlying cause of CF.

Sionna is developing a pipeline of potential modulators that could benefit people with the most common CF-causing mutation, F508del, and may eventually offer an alternative to currently approved CFTR modulators. The novel modulators target NBD1, the region of the gene where the F508del mutation occurs. No modulator currently on the market targets NBD1.

The announcement comes after more than a decade of supporting early-stage F508del CFTR corrector discovery work at Genzyme/Sanofi that would eventually lead to Sionna’s current modulator pipeline. Since 2011, the Foundation has provided more than $38 million to this research, including today’s $5 million Series B investment in Sionna.

“We continue to support Sionna's novel approach in addressing the underlying cause of cystic fibrosis because we are committed to developing additional therapeutic options for people living with cystic fibrosis,” said Michael Boyle, MD, president and CEO of the Cystic Fibrosis Foundation. “Today's announcement represents a step toward all people living with CF having access to an effective treatment for their disease, including those unable to tolerate current therapeutic options.”

Our Approach to Innovation

The CF Foundation's established venture philanthropy model provides early-stage funding to companies to develop breakthrough treatments for adults and children with cystic fibrosis. This approach has super-charged the field of CF research by helping to de-risk and accelerate the development of new technologies in cystic fibrosis, resulting in more than 16 approved therapies – including four therapies that address the underlying cause of CF for many people with the disease. However, critical work remains to ensure that every person has an effective treatment to address the root cause of their disease, regardless of mutation.

Today, the Foundation is investing more aggressively than ever before to support the development of the next generation of transformative therapies to help people with CF live their longest, healthiest lives possible.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.