Although adeno-associated virus (AAV) gene therapies have represented a large swath of the gene therapeutic landscape for years, they have been weighed down by various limitations and adverse effects. The biotech Carbon Biosciences, led by seeks to disrupt the field of cystic fibrosis gene therapies by introducing new viral vector to the mix, bocavirus. This move is made as part of a new $38 million partnership with the Cystic Fibrosis Foundation.
According to Kyle LaHucik, “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.”
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(Source: Endpoints News, June 21st, 2022)