A biotech startup launches with $38M to develop a cystic fibrosis gene therapy

Dive Brief:

• Carbon Sciences, a Massachusetts-based startup, launched Tuesday with $38 million in funding to develop a gene therapy for cystic fibrosis.
• The Series A financing is led by investment firm Agent Capital, which was joined by the venture arm of Japanese drugmaker Astellas as well as the Cystic Fibrosis Foundation and the Longwood Fund, which in 2020 partnered to fund research into new cystic fibrosis therapies.
• Cystic fibrosis, a genetic disease that damages the lungs, is treatable for most patients with effective drugs developed by Vertex Pharmaceuticals. About 10% of patients aren’t helped by those therapies, however, spurring drugmakers like Carbon to explore alternative treatment approaches.

Dive Insight:

Vertex’s latest treatment for cystic fibrosis is a “triple combination” drug called Trikafta, which like others from Vertex boosts the activity of a protein that’s defective in people with the disease. It has quickly become a go-to treatment, entrenching Vertex’s dominant market position.

Other companies have tried to create competing treatments that can match Vertex’s medicines, but have so far fallen short.

Still, even Trikafta doesn’t work for a minority of patients, leaving an opportunity for other developers. Carbon is one, and has plans to develop an unorthodox gene therapy solution.

Gene therapies often rely on what are known as adenovirus-associated viruses, or AAVs, to transport genetic material into the body’s cells. However, AAVs have limitations and not all types work well outside of organs like the liver or eye. Carbon is instead turning to parvoviruses in an attempt to reach less accessible tissues. The company claims parvoviruses also can carry bigger genes and could potentially be administered multiple times.

According to Carbon CEO Joel Schneider, parvoviruses have a high affinity for the lung — something he said in an interview has not “been demonstrated by AAV-based approaches.”

Vertex is also looking at genetic medicines to reach the last 10% of cystic fibrosis patients, and has partnerships with gene editing companies CRISPR Therapeutics, Arbor Biotechnologies and Affinia Therapeutics.

While Carbon’s $38 million raise is relatively small, Schneider said Carbon will remain “opportunistic to the funding climate.” Recently, the biotech sector has been weathering one of the worst downturns in recent history, with sharp declines in public offerings and knock-on effects to biotech funding.

The Cystic Fibrosis Foundation, which funded some of Vertex’s research and has shown interest in new treatment technologies, chipped in $6 million of the Series A funding. The foundation is also allowing Carbon to use its laboratories in Lexington until the company can find its own space.

The funding will help Carbon push the cystic fibrosis program toward the clinic, as well as aid the startup’s efforts to identify viruses for gene therapy delivery.

Carbon’s approach builds on work by John Engelhardt, director of the University of Iowa’s Center for Gene Therapy of Cystic Fibrosis, and Robert Kotin, who is a professor of microbiology and physiological systems at the University of Massachusetts Chan Medical School.

Both Engelhardt and Kotin hold positions with Carbon. Kotin is founding its chief scientific officer and Engelhardt has joined the company’s scientific advisory board.

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