Vertex and Moderna collaborate in rare disease therapy using gene editing

Pharma News: Moderna and Vertex Pharmaceuticals announced a new strategic research collaboration and licensing agreement aimed at the discovery and development of lipid nanoparticles (LNPs) and mRNAs for the delivery of gene-editing therapies for the treatment of cystic fibrosis (CF).

Cystic Fibrosis (CF) is an inherited condition that affects many organs in the
body. The organs most affected are the lungs and gut. Every four days a baby is born in Australia with cystic fibrosis (CF) and more than one million Australians are carriers of cystic fibrosis gene.

The three-year research collaboration initially will focus on the discovery and optimization of novel LNPs and mRNAs that can deliver gene-editing therapies to cells in the lungs, enabling functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to be produced.

“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, Chief Executive Officer 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.”

“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% of patients do not produce any CFTR protein and so are unlikely to benefit from our existing medicines.

“Over the past 5 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,” said David Altshuler, M.D., Ph.D., Vertex’s Executive Vice President, Global Research and Chief Scientific Officer.

“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,” he added.

Under the terms of the agreement, Moderna will conduct research activities to discover and optimise novel LNPs for the delivery of gene-editing therapies to lung cells for the treatment of CF. Moderna will receive $75 million upfront and will be eligible to receive up to $380 million in development, regulatory and commercial milestones, plus tiered royalties on any products that result from the collaboration. Moderna will be responsible for the discovery and manufacturing of LNPs and mRNA constructs encoding gene-editing endonucleases.

Vertex will be responsible for providing other components of the gene-editing therapies to be formulated into LNPs, as well as subsequent preclinical and clinical development and potential commercialisation efforts.

This new collaboration adds to the existing relationship between Moderna and Vertex in their recently extended collaboration aimed at the discovery and development of mRNA therapeutics for the treatment of CF.