New Biotech Company to Develop Lung-specific CF Gene Therapy Vectors

A newly launched biotech company called Carbon Biosciences is aiming to develop lung-specific gene therapy vectors — vehicles to deliver genetic material directly into a cell — to help treat cystic fibrosis (CF).

The company has $38 million in series A financing, funded in part by the Cystic Fibrosis Foundation.

“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,” Joel Schneider, PhD, Carbon’s president and CEO, said in a press release.

“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,” Schneider said.

CF is caused by mutations in the CFTR gene. The general aim of gene therapy is to deliver a healthy copy of this gene to the body’s cells.

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Most gene therapies deliver their genetic cargo to cells via the use of a viral vector. The adeno-associated virus (AAV), for instance, has often been used in gene therapy development.

The new company’s platform aims to develop vectors for gene therapy using another type of virus called parvovirus.

Parvoviruses are a family of small viruses that infect many warm-blooded animals. These viruses are perhaps best known for causing disease in dogs — specifically, canine parvovirus or “parvo.” In humans, a parvovirus is also responsible for fifth disease, a mild condition causing rash that’s more common in children than adults.

According to the company, Carbon’s lead parvovirus-based gene therapy candidate — dubbed CGT-001 — is notable because it has demonstrated tissue tropism to the lungs. That means the viral vector will preferentially deliver its genetic payload to lung cells. This could be especially excellent in CF lungs, where the organ is often the most affected.

“Carbon’s novel platform addresses key challenges with AAV and non-viral based therapies,” said John Engelhardt, PhD, scientific co-founder of Carbon, and director of the Center for Gene Therapy at the University of Iowa.

“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,” Engelhardt said. “Preliminary pre-clinical data as well as studies on human populations suggest wide applicability of our lead clinical candidate and the potential to re-dose patients.”

Financing for the new company’s launch — expected to advance its CF program toward clinical testing — was led by the healthcare venture capital firm Agent Capital.

“Identifying that can effectively deliver therapeutics to target tissues, such as the lung, has been a major challenge in realizing the full potential of gene target tissues,” said Geeta Vemuri, Ph.D., 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 technology will enable a diversified pipeline with potential applications across the wide range of tissues impacted in many unaddressed diseases,” Vemuri added.

The CF Foundation contributed $6 million to the financing, through its collaboration with the venture capital firm Longwood Fund, as part of the foundation’s Path to a Cure initiative. Early scientific work for Carbon was performed at the CF Foundation Therapeutics Lab in Massachusetts, marking the first time the foundation has hosted researchers from a startup company.

“Being able to support Carbon Biosciences in its early stage research both financially and at our lab is an incredible step in evolving our investment strategy to attract the best science and technology to the CF space,” Martin Mense, PhD, senior vice president of drug discovery at the CF foundation and head of the CF Foundation Therapeutics Lab, said in a separate press release.

“Their unique scientific approach combined with our ability to help support their understanding of the challenges specific to CF biology could prove to be a giant step toward addressing the unmet need of innovative treatments in cystic fibrosis. It is our hope that they are the first of many companies we are able to incubate in the future,” Mense added.

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