19 April 2022 - - US-based non-profit organization The Cystic Fibrosis Foundation has invested USD 5m in US-based life science startup Sionna Therapeutics, a company founded in 2019, the group said.

The company is focused exclusively on developing cystic fibrosis transmembrane conductance regulator 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 USD 38m to this research, including TODAY's USD 5m series B investment in Sionna.

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.

The Cystic Fibrosis Foundation is 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, the group said.

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.