Cystic Fibrosis Start-Up Raises $111M in New Funds

(National Heart, Lung, and Blood Institute, NIH)

19 Apr. 2022.  A developer of new treatments for the inherited lung disease cystic fibrosis emerged from stealth mode, raising $111 million in new venture funds. One of the investors in Boston-based Sionna Therapeutics, the Cystic Fibrosis Foundation, is also sponsoring a challenge competition for start-ups creating genetic therapies for the disease.

Sionna Therapeutics is a three year-old enterprise that up to now operated in private. The company says it’s developing therapies that address an underlying cause of cystic fibrosis, an inherited disorder of glands that produce mucus in the lungs and airways. Mucus keeps lungs and airways moist, and also helps prevent infections, but because of mutations passed from parents to children, mucus can become thick and sticky, and build up in the lungs and airways. The build-up of mucus can also affect the pancreas, liver, intestines, and other organs.

Because of accumulated mucus, bacterial infections are easier to form in the lungs, making cystic fibrosis a debilitating disease. Cystic Fibrosis Foundation says the disease affects some 70,000 people worldwide, of which 30,000 are in the U.S. The disease is traced to alterations in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene that normally makes thinner and more flowing mucus, but is missing or defective in people with the disease.

Target considered undruggable

The most common CFTR mutation linked to cystic fibrosis is called deletion of phenylalanine 508 or delta F508. This mutation destabilizes CFTR to where it loses its regulatory functions allowing mucus to build up in the lungs and airways. Current cystic fibrosis or CF therapies, says Sionna, treat symptoms of the disease or correct only some of the functional loss from delta F508 mutations. The company says its technology produces small molecule therapies that target a primary binding domain in the mutated CFTR protein called NBD1 where the delta F508 chemistry resides. Sionna says its treatments stabilize the NBD1 binding domain, enabling CFTR to produce normal lung mucus.

“NBD1 is a well-known and researched target, but it has been considered undruggable until now,” says Sionna Therapeutics president and CEO Mike Cloonan in a company statement. “Based on our focused efforts and continued progress on NBD1, we see the potential to normalize CFTR function in the vast majority of people with CF.”

Sionna Therapeutics is raising $111 million in its second venture funding round, led by life science venture investor OrbiMed Advisors in New York. Joining the round are Sionna’s earlier investors RA Capital, The Rise Fund, Atlas Venture, and the Cystic Fibrosis Foundation, with new investors T. Rowe Price Associates and Q Healthcare Holdings, a subsidiary of QIA, the sovereign wealth fund of Qatar. The company says it raised $39 million in earlier rounds.

Golden Ticket challenge

Cystic Fibrosis Foundation says it contributed $5 million to Sionna Therapeutics’ financing. The foundation is also sponsoring a challenge competition called Golden Ticket to encourage researcher-entrepreneurs in gene therapies, delivery, and editing to develop the next generation of cystic fibrosis treatments. The competition is open to new businesses still in seed or first (series A) funding rounds developing genetic therapies for cystic fibrosis. Companies can be affiliated with university labs or independent.

The Golden Ticket challenge will award three companies a year of free lab and office space at Bakar Labs, a life science business incubator affiliated with University of California in Berkeley. Winners of the competition will also gain access to Cystic Fibrosis Foundation research resources, including its cell and sample collections, patient registry, and network of 90 clinical trial sites in the U.S.

“As part of our relentless pursuit of a cure, we are dedicated to investing in early stage science,” says the foundation’s vice president for drug discovery Martin Mense in a statement, “We know that to make progress toward our bold mission to cure CF, we need to attract bold, like-minded researchers.”

Golden Ticket entries are accepted from 2 May to 30 May 2022, with finalists notified by 29 June. Finalists are invited to present their entries and pitches to a panel of judges on 20 July, with winners announced after the pitch session.

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