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Biotechs Partner on Gene-Editing Cystic Fibrosis Therapy

lung illustration

(Kai Stachowiak, Pixabay)

17 Sept. 2020. Two biotechnology companies are adding to their current collaboration to develop delivery techniques for gene-editing treatments for cystic fibrosis. The partnership with Vertex Pharmaceuticals Inc. in Boston could bring Moderna Inc. in Cambridge, Massachusetts as much as $455 million if all phases of the collaboration are fulfilled.

Cystic fibrosis or CF is an inherited disease of the glands that make mucus, a substance keeping the lungs and airways moist, as well as helping prevent infection. The disease results from mutated genes passed from each parent to their children. With cystic fibrosis, the mucus becomes thick and sticky, and builds up in the lungs and airways. The build-up of mucus can also affect the pancreas, liver, intestines, and other organs.

The accumulation of mucus makes it easier for bacteria to grow, leading to repeated lung infections. The build-up of mucus in the pancreas can likewise block ducts in the pancreas, interrupting the flow of enzymes for digestion. The disease is traced to mutations in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene that makes the normal thinner, more flowing mucus, but is missing or defective in people with the disease.

Cystic fibrosis is a major focus for Vertex, which already has four cystic fibrosis drugs approved by FDA. “Vertex’s CFTR modulator therapies have the potential to treat the vast majority of CF patients and address the underlying cause of their disease, says Vertex’s chief scientist David Altshuler in a statement. “However, approximately 10 percent of patients do not produce any CFTR protein and so are unlikely to benefit from our existing medicines. Over the past five years, we have made important progress in our research efforts aimed at the creation of genetic therapies for CF, with the delivery of such therapies remaining the most significant technological and scientific challenge.”

Vertex and Moderna are already collaborating on cystic fibrosis therapies. In a 2016 deal, as reported by Science & Enterprise, Vertex is licensing Moderna’s messenger RNA technology to provide functioning CFTR proteins for people with cystic fibrosis. Under that deal, companies are collaborating on new treatments for cystic fibrosis, but also for delivery of messenger RNA into the lungs.

The new agreement focuses more on delivery of genetic therapies for cystic fibrosis. The deal calls for Moderna to discover, optimize, and later manufacture lipid nanoscale particles with messenger RNA to deliver gene-editing enzymes into the lung cells of cystic fibrosis patients. In addition to its work with messenger RNA, Moderna also develops engineered lipid nanoparticles for delivery of biologics, including its vaccines. Vertex will provide gene-editing components for the therapies, and conduct preclinical research, clinical trials, and commercialization steps.

Moderna is receiving $75 million as an initial payment from Vertex, with up to $380 million in development, regulatory, and commercial milestones, as well as royalties on new products from the collaboration. “Our first collaboration with Vertex to deliver mRNA coding for cystic fibrosis protein in lung cells is advancing well,” notes Moderna CEO Stéphane Bancel, “and this second collaboration aims at using Moderna’s technologies to explore the use of gene editing in lung cells.”

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