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RNA Technology Licensed for Cystic Fibrosis Therapy

RNA molecule illustration

RNA molecule illustration (Nicolle Rager Fuller, National Science Foundation)

6 July 2016. Two biotechnology companies are collaborating on treatments for cystic fibrosis that use genetic material to address underlying causes of the inherited disease. The 3-year deal with Vertex Pharmaceuticals is expected to bring Moderna Therapeutics as much as $315 million.

Cystic fibrosis 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.

Vertex Pharmaceuticals, in Boston, is a biotechnology company developing drugs for a variety of disorders including cystic fibrosis, cancer, influenza, pain, and spinal cord injury. The company has 2 drugs already approved for cystic fibrosis — Orkambi and Kalydeco — and 4 other drugs in development for the disease, including a compound in late-stage clinical trials

Moderna Therapeutics in Cambridge, Massachusetts, develops medications that use messenger RNA, a nucleic acid related to DNA employed by cells to produce the amino acids in proteins for carrying out bodily functions. Moderna designs what it calls modified messenger RNA to produce proteins that act like drugs as treatments for diseases, creating therapeutic antibodies in less time and lower cost than current genetic engineering methods.

Vertex’s drugs for cystic fibrosis address functioning of 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. The deal calls for Vertex to license Moderna’s messenger RNA technology to provide functioning CFTR proteins for people with cystic fibrosis. The companies will collaborate on research and development of new treatments, including delivery of messenger RNA into the lungs.

The agreement gives Vertex an exclusive license for Moderna’s messenger RNA applied to treatments for cystic fibrosis that generate functioning CFTR proteins. Vertex is expected to lead all preclinical, development, and commercialization work between the two companies and fund all research expenses.

Vertex is providing Moderna an initial $20 million payment, as well as a $20 million convertible note, a loan that can convert later into an equity investment. In addition, Moderna will be eligible for $275 million in future development and regulatory milestones, as well as royalties on sales of products from the collaboration.

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