Moderna, Vertex Team Up to Develop Gene-editing Delivery Techniques

Moderna Therapeutics and Vertex Pharmaceuticals have entered a three-year research and licensing agreement aimed at developing gene-editing delivery techniques for the treatment of cystic fibrosis (CF).

While Moderna will focus on ways to deliver gene-editing therapies to lung cells, Vertex will concentrate on other gene-editing components — and on subsequent preclinical and clinical development studies and potential commercialization efforts.

Specifically, Moderna’s goal is to discover and optimize lipid nanoparticles (LNPs) for the delivery of messenger RNA (mRNA) constructs that encode gene-editing endonucleases — proteins that are able to cut DNA.

LNPs are made of fatty molecules called lipids that can cross the cell membrane, which is inaccessible to mRNA.

“Vertex’s CFTR modulator therapies have the potential to treat the vast majority of CF patients and address the underlying cause of their disease. However, approximately 10 percent of patients do not produce any CFTR protein and so are unlikely to benefit from our existing medicines,” David Altshuler, MD, PhD, Vertex’s executive vice president, global research and chief scientific officer, said in a press release.

“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,” Altshuler said.

This new collaboration to develop such gene-editing delivery techniques follows a previous deal signed in 2016. That partnership centered on developing mRNA-based therapies to enable cells to produce functional cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is normally disrupted in CF.

“We are pleased to enter into this second collaboration with Vertex aimed at delivering potentially novel treatments for patients with cystic fibrosis using gene editing,” said Stéphane Bancel, CEO of Moderna.

“Our first collaboration with Vertex to deliver mRNA coding for cystic fibrosis protein in lung cells is advancing well and this second collaboration aims at using Moderna’s technologies to explore the use of gene editing in lung cells,” Bancel added.

Under the terms of the agreement, Moderna will receive $75 million as an initial payment from Vertex. Moderna will be eligible for an additional $380 million in development, regulatory and commercial milestones, and will receive royalties on any products that result from the collaboration.

“The combination of Moderna’s unique expertise in the discovery and manufacturing of novel LNP delivery systems and mRNA technologies, combined with Vertex’s scientific, clinical and regulatory capabilities in CF, will accelerate the development of groundbreaking genetic therapies for people with CF and supports our commitment to developing therapies for all people living with CF,” Altshuler said.

Of note, Vertex currently has four FDA-approved medicines for CF on the market.