Carbon Biosciences Launches with $38 Million to Advance Novel Gene Therapy Platform and Pipeline

Carbon Biosciences raised $38 million in a series A financing to advance the development of novel parvovirus-derived gene therapies.

Photo: Joel Schneider, president and CEO of Carbon Biosciences

Agent Capital led the financing and was joined by Longwood Fund, which founded Carbon, Astellas Venture Management, the Cystic Fibrosis Foundation, Solasta Ventures, University of Tokyo Innovation Platform (UTokyoIPC), and Camford Capital.

Carbon is developing a novel parvovirus-based vector platform with demonstrated in vivo tissue specificity, increased cargo capacity, and minimal natural neutralizing immunity, with the potential to widen patient applicability and enable gene therapy re-administration.

The company plans to use the series A funding to advance the development of its programs for genetic diseases, initially building on the groundbreaking research of scientific co-founders John Engelhardt, director of the Center for Gene Therapy at the University of Iowa, and Robert Kotin, professor of Microbiology and Physiological Systems at the University of Massachusetts Chan Medical School.

Carbon’s lead gene therapy candidate, CGT-001, targets lung tissue with the capacity to deliver the full length CFTR gene and an appropriate promoter with demonstrated upper airway tissue tropism and repeatable dosing in gold-standard cystic fibrosis models.

Cystic fibrosis is an inherited condition caused by mutations in the CFTR gene and is inherited in an autosomal recessive fashion. It causes mucus to build up and clog some of the organs in the body, particularly in the lungs and pancreas. Over time, mucus buildup and infections can lead to permanent lung damage, including the formation of scar tissue (fibrosis) and cysts in the lungs. Mucus can also block the digestive tract and pancreas, leading to digestive problems.

“Carbon’s platform has the potential to expand the role of gene therapy in treating some of the world’s most devastating and difficult to treat diseases,” said Joel Schneider, president and CEO of Carbon Biosciences. “Our vision is to enable a new generation of genetic medicines with differentiated vectors that have the potential to address the immunological, targeting and payload limitations inherent in current viral and non-viral delivery technologies. As the first jointly funded program launched under a collaborative agreement between the Cystic Fibrosis Foundation and Longwood Fund, we are motivated by the potential impact our research may have in significantly improving care for cystic fibrosis patients.”

Geeta Vemuri, managing partner and founder of Agent Capital said that 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.

“We believe that Carbon’s proprietary platform can address this challenge by leveraging novel vectors from the broader parvovirus family to deliver optimal payloads to specific tissues,” she said. “Carbon’s technology will enable a diversified pipeline with potential applications across the wide range of tissues impacted in many unaddressed diseases.”

In addition to Kotin and Engelhardt, Carbon’s co-founders are David Steinberg, who served as founding CEO (Longwood Fund); Jianming Qiu, (University of Kansas); Ziying Yan, (University of Iowa); Sebastian Aguirre, (Carbon Biosciences); and Lucy Liu, (Longwood Fund).

Author: Rare Daily Staff