Gene Therapy Start-Up Gains $38M in Early Funds

(Genome Research Limited, Flickr)

21 June 2022. A new company developing genetic treatments with a high-capacity engineered virus to deliver its therapeutic payloads is raising $38 million in venture funds. Carbon Biosciences in Lexington, Massachusetts is based on research at University of Iowa and University of Massachusetts medical schools, and founded last year by Longwood Fund in Boston.

Carbon Biosciences is creating gene therapies using paroviruses to deliver genetic materials to target cells and tissue. One form of parovirus can infect humans causing a mild rash called fifth disease that mainly affects children, while other forms affect household pets. Robert Kotin, a microbiologist at UMass medical school in Worcester and one of Carbon Bio’s scientific founders, studies paroviruses and reported on their potential for delivering larger and more complex gene therapies than adeno-associated viruses used in many of today’s gene therapies. Kotin is also Carbon Bio’s chief scientist.

Another of Carbon Bio’s scientific founders is John Englehardt, a cell biologist at University of Iowa in Iowa City and director of the school’s gene therapy center. Englehardt and associates study gene therapies to treat cystic fibrosis, a disease of glands producing mucus in the lungs and airways. In its normal state, mucus keeps lungs and airways moist, and also helps prevent infections, but in some cases genetic mutations passed from parents to children can create thick and sticky mucus that builds up in lungs and airways. Because of this accumulated mucus, bacterial infections are easier to form in the lungs, making cystic fibrosis a debilitating disease.

Deliver a full-length gene

In a paper published last year, Englehardt and a colleague outlined the limits of adeno-associated viruses in delivering gene therapies for cystic fibrosis, particularly their inability to transfer the full cystic fibrosis transmembrane conductance regulator or CFTR gene needed to replace mutated genes in people with the disease. That transfer vehicle, noted the authors, needs to deliver a treatment with high tropism or payload capacity to deliver the full healthy gene and avoid multiple doses of partial gene segments.

Carbon Bio’s first gene therapy candidate, code-named CGT-001, is a genetic treatment for cystic fibrosis using an engineered parovirus as the delivery vector. Because of the parovirus’s larger capacity, the company believes its technology can address limitations of adeno-associated viruses with gene therapies for this disease. “Our lead program,” says Englehardt in a company statement released through BusinessWire, “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.” Englehardt, an advisor to Carbon Bio, adds that “pre-clinical data as well as studies on human populations suggest wide applicability of our lead clinical candidate ….”

Longwood Fund founded Carbon Biosciences last year, and is taking part in the company’s first venture round raising $38 million. Leading the round is Agent Capital, a health care venture investor in Waltham, Mass. Also taking part in the financing are Cystic Fibrosis Foundation, Solasta Ventures, University of Tokyo Innovation Platform, Astellas Venture Management, and Camford Capital. Cystic Fibrosis Foundation supports start-ups developing treatments both with venture investments and, as reported by Science & Enterprise in April 2022, a challenge competition for new genetic therapies.

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