Carbon Biosciences emerges with a new viral vector to shake up the cystic fibrosis gene therapy field
Carbon Biosciences wants to disrupt the field of AAV gene therapy by tapping into a family of thousands of viruses to find ones that allow for the “next generation of viral delivery vehicles,” as CEO Joel Schneider puts it.
The Massachusetts biotech emerged Tuesday with $38 million in partnership with the Cystic Fibrosis Foundation to take a bocavirus-based — part of the broader parvovirus web — gene therapy into the clinic for the lung disorder.
Adeno-associated virus gene therapies have tripped up the biotech R&D world for years now, with various toxicity issues and other limiting factors, so Carbon wants to go after viruses that allow for gene therapies to be delivered more than once, which will open up the opportunity to target more patients.
The startup’s first therapy, named CGT-001, can be delivered more than once, specifically targets the lung tissue and is able to transport the full-length cystic fibrosis gene, Schneider tells Endpoints News.
The lead candidate is also able to carry 30% to 40% more than the typical AAV, which Schneider knows a thing or two about after eight years in the C-suite at AAV-based Solid Biosciences, which is attempting to tackle Duchenne muscular dystrophy.
He declined to say when Carbon will enter the clinic, but noted the Series A will get the startup there. The first human trial will target CF patients who don’t have a modulator therapy, Schneider said.
Carbon is the first startup launched out of the 2020 incubator created between Longwood Fund and the Cystic Fibrosis Foundation.
Carbon is based on science from John Engelhardt, director of University of Iowa’s Center for Gene Therapy of Cystic Fibrosis, and Robert Kotin, professor of microbiology and physiological systems at University of Massachusetts Chan Medical School.
Lucy Liu, VP and co-founder at Carbon and a senior associate at Longwood, recalled calling Engelhardt at the end of 2020 after the Longwood-CFF program launched and at the end of this conversation, he mentioned he’d been working on a way to overcome AAV’s limitations of pre-existing immunity, small cargo capacity, limited tissue tropism, etc.
Then, serendipitously within a month, Kotin came to Longwood and CFF saying he’d been working on a similar idea in his lab. Kotin is now interim CSO of Carbon.
So in mid-2021, Carbon was formed and has been working out of CFF’s labs in Lexington, MA. The startup will open its own lab in Waltham, MA, by year’s end, Schneider said.
The story behind the startup’s name highlights the bold ambition Carbon is on.
“We’re really allowing evolution and virology to take the front seat here, and so carbon, which is the building block of all life, we’re really allowing that to drive our approach where we’re identifying already existing viruses and taking the best part of what they’ve evolved and what they’ve offered and turn that into a viral vector for us to use for therapeutic processes,” Liu said.
Aside from getting into that first human study, Carbon is building out its platform by identifying other parvoviruses to use to target other tissues, Schneider said. He’s also focused on nearly doubling the team from 13 people today to almost 25 by the end of 2022.
Down the line, the startup could go beyond gene therapies and into gene editing.
“Initially, Carbon Bio will pursue its own therapeutic development, but collaboration with another Foundation-funded company, e.g., for the delivery of a gene-editing therapy is absolutely part of future considerations,” CFF’s SVP of drug discovery, Martin Mense, told Endpoints in an email.
The Series A was led by Agent Capital and included backing from Longwood, Astellas Venture Management, Solasta Ventures, University of Tokyo Innovation Platform, Camford Capital and the CFF. Adicet Bio president and CEO Chen Schor is board chair.