RARE Daily

Moderna Targets Rare Disease with Two Collaborations

September 17, 2020

Marie Daghlian

Moderna, currently known for its efforts to develop an mRNA-based vaccine targeting the SARS-COVID 19 virus, has struck separate deals with Vertex Pharmaceuticals and Chiesi Farmaceutici focused on mRNA therapies for rare diseases.

Moderna’s agreement with Vertex is the second deal between the partners focused on developing genetic therapies for cystic fibrosis (CF), an area where Vertex is already a leader in therapies targeting the rare, progressive, and life-shortening genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. While there are many different types of CFTR mutations that can cause the disease, most people with CF have at least one F508del mutation.

The partners’ three-year research collaboration, valued at up to $455 million, is aimed at the discovery and development of lipid nanoparticles (LNPs) and mRNAs for the delivery of gene-editing therapies, initially to the lungs to enable the production of functional cystic fibrosis transmembrane conductance regulator (CFTR) protein.

Under the terms of the agreement, Moderna will conduct research activities to discover and optimize novel LNPs for the delivery of gene-editing therapies to lung cells for the treatment of CF. Vertex will pay Moderna $75 million upfront and 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 commercialization efforts.

“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. 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, executive vice president of global research and chief scientific officer at Vertex. “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.”

Moderna’s collaboration with Chiesi Farmaceutici, valued at up to $425 million, is focused on pulmonary arterial hypertension (PAH), a rare progressive disorder characterized by high blood pressure in the arteries of the lungs with concomitant right heart failure.

Under the terms of their agreement, Moderna will lead discovery efforts, leveraging its mRNA technology and delivery platforms along with Chiesi’s expertise in the field of PAH biology. Chiesi will lead development and worldwide commercialization activities and will fund all expenses related to the collaboration. Moderna will receive $25 million upfront and is eligible for more than $400 million in development, regulatory, and commercial milestones, as well as tiered double-digit royalties on net sales.

“This new relationship continues our commitment to partner with companies that have unique expertise in serious diseases, and who share our vision of using mRNA therapeutics to address the unmet needs of patients,” said Stephen Hoge, president of Moderna.

Photo: Stephen Hoge, president of Moderna


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