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Beyond Coronavirus: Moderna Teams Up With Vertex on Cystic Fibrosis Therapy

By Brian Orelli, PhD – Sep 17, 2020 at 7:03PM

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This is the companies' second project to tackle the lung disease.

All eyes are on Moderna's (MRNA -0.55%) coronavirus vaccine, but the biotech is looking ahead, setting up a new research collaboration with Vertex Pharmaceuticals (VRTX -1.01%) to develop a treatment for cystic fibrosis, which is Vertex's specialty.

This is actually the companies' second collaboration to tackle cystic fibrosis. The first project involved using Moderna's messenger RNA (mRNA) technology to express the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is mutated in patients with cystic fibrosis.

The newest project seems a little more ambitious, but could be a one-time treatment for cystic fibrosis. Instead of expressing CFTR from mRNA, the companies plan to use Moderna's messenger RNAs delivered with its lipid nanoparticles (LNPs) to express gene-editing proteins that would edit the patients' DNA so the lung cells express functional CFTR protein.

Doctor with a stethoscope on a patient's chest.

Image source: Getty Images.

The three-year research deal calls for Vertex to pay $75 million upfront to Moderna, which is also eligible for up to $380 million in development, regulatory, and commercial milestone payments, as well as tiered royalties on sales of any products that result from the collaboration.

Moderna will be responsible for manufacturing the LNPs and mRNA, while Vertex will contribute other components of the gene-editing therapies that will be formulated into LNPs. Once a candidate is determined, Vertex will be responsible for preclinical and clinical development, as well as selling the therapy, should it make it onto the market.

Interestingly, Vertex already has a license option on CRISPR Therapeutics (CRSP -2.68%) gene-editing program to treat cystic fibrosis. It appears Vertex is hedging its bets on two fronts with this second collaboration with Moderna.

Brian Orelli, PhD has no position in any of the stocks mentioned. The Motley Fool owns shares of and recommends CRISPR Therapeutics. The Motley Fool recommends Vertex Pharmaceuticals. The Motley Fool has a disclosure policy.

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Stocks Mentioned

Vertex Pharmaceuticals Incorporated Stock Quote
Vertex Pharmaceuticals Incorporated
$283.45 (-1.01%) $-2.89
Moderna, Inc. Stock Quote
Moderna, Inc.
$123.64 (-0.55%) $0.68
CRISPR Therapeutics Stock Quote
CRISPR Therapeutics
$63.16 (-2.68%) $-1.74

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