Carbon Biosciences launches with $38m Series A financing round to advance cystic fibrosis gene therapy candidate to clinic

By Jane Byrne

- Last updated on GMT

© GettyImages/Dr_Microbe
© GettyImages/Dr_Microbe

Related tags vector Gene therapy Cystic fibrosis

The developer says it is harnessing parvovirus vectors that can deliver larger gene therapy payloads with enhanced tissue specificity and with minimal neutralizing immunity.

Carbon Biosciences has been founded by the Longwood Fund and gene therapy pioneers, John F Engelhardt, director, Center for Gene Therapy at the University of Iowa, and Robert M Kotin, professor of microbiology and physiological systems at the University of Massachusetts Chan Medical School.

It is looking to expand the gene therapy toolbox for the treatment of the world’s most devastating and difficult to treat diseases.

The $38m Series A financing round, led by Agent Capital, is targeted at Carbon’s lead cystic fibrosis program, advancing it towards the clinic as well as supporting the development of its proprietary platform.

Also participating in that investment round was Longwood Fund, Astellas Venture Management LLC, the Cystic Fibrosis Foundation, Solasta Ventures, University of Tokyo Innovation Platform (UTokyoIPC), and Camford Capital.

Joel Schneider, CEO of Carbon Biosciences, said the company’s 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.”

Carbon’s lead candidate, CGT-001, targets lung tissue with demonstrated upper airway tissue tropism and repeatable dosing in gold-standard cystic fibrosis models.

Realizing the full potential of gene therapy

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, according to Geeta Vemuri, managing partner and founder of Agent Capital. “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."

Carbon’s novel platform addresses key challenges with AAV and non-viral based therapies, outlined Engelhardt. “Our lead program is the first gene therapy program demonstrating tissue tropism to the lung with the capacity to deliver the full length CFTR gene and an appropriate promotor.”

Preliminary pre-clinical data as well as studies on human populations suggest wide applicability of that investigation therapy and the potential to re-dose patients, he added.

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