Carbon Biosciences exits stealth with $38M, unveils cystic fibrosis gene therapy candidate

Carbon Biosciences has emerged from stealth mode with $38 million in series A funds to push its cystic fibrosis candidate into the clinic and build out its gene therapy platform.

The biotech's cystic fibrosis work is underpinned by a collaboration between the Cystic Fibrosis Foundation and Carbon's founder, the healthcare-focused venture capital firm Longwood Fund. Vertex Pharmaceuticals currently has four therapies approved for the disease—with its first approval for Kalydeco back in 2012—but they are treatments for specific patient groups. While Roche has also maintained a foot in the market, there remains no cure for the inherited lung disorder.

Carbon’s parvovirus-based vector platform is designed to deliver larger gene therapy payloads with high tissue specificity and minimal neutralizing immunity. Both the platform and the biotech's lead candidate CGT-001 offer re-dosing potential, with CGT-001 able to target upper airway tissue in cystic fibrosis models. 

The series A funding round was led by Agent Capital, an international life sciences firm that invests in healthcare companies targeting unmet patient needs, including recent support for immunity specialist Imcheck earlier this month.

“Identifying vectors that can effectively deliver therapeutics to target tissues, such as the lung, has been a major challenge in realizing the full potential of gene therapy,” Geeta Vemuri, Ph.D., managing partner and founder of Agent Capital, and a new addition to Carbon’s board of directors, said in a June 21 release. Carbon's platform could tackle this problem by using new vectors from the parvovirus family of viruses to deliver optimal payloads to specific tissues, according to Vemuri, who added that Carbon’s tech also has application potential across a range of tissues impacted in several other unaddressed diseases.

Longwood Fund and the Cystic Fibrosis Foundation were joined in the series A round by investors Astellas Venture Management, Solasta Ventures, University of Tokyo Innovation Platform and Camford Capital. In tandem with the financing round, Joel Schneider, Ph.D, has stepped on as Carbon president and CEO, leaving behind his role as chief operating officer at Solid Biosciences, where he was the biotech’s first employee.  

Carbon's founder Longwood Fund has a proven history in both setting up and contributing to the creation of biotechs addressing unmet medical needs, including the likes of Vertex, Acceleron and Alnylam. In May, the VC firm pumped funds into engineered B cell platform developer Be Bio as well as Rectify Pharmaceuticals, which touts its ABCs—three letters that signify a group of proteins with mutations that destroy functions in multiple organs.