Unlocking the Power of Genetics to Transform Lives
Welcome to Moderna Genomics, a new research endeavor of Moderna.
When Moderna was founded, at our core was a very simple and profound idea. We knew that we could change the way medicine is made by using mRNA to teach the body to make its own medicine.
But our innovation doesn’t end there.
Now, Moderna Genomics is tackling our next “blue sky challenge” — to develop individualized medicines that could provide benefits for patients with genetic disease.
Delivering Individualized Genetic Medicines with mRNA
The goal of medicines that are potentially curative is to treat the root cause of disease. Individualized genetic medicines aim to correct pathogenic mutations.
Moderna Genomics is researching the unique advantages of Moderna’s mRNA platform as a vehicle to deliver gene-editing medicines. We are working to develop tailor-made systems targeting genetic mutations specific to a patient’s genome.
We are advancing beyond the traditional one-size-fits-all approach to medicine with the goal of creating one therapy for one patient – or “n-of-one” individualized genetic treatments.
Pioneering Individualized Therapies for Monogenic Liver Diseases
Millions of people suffer from diseases caused by errors or mutations in a single gene (monogenic diseases). In fact, there are ~10,000 monogenic diseases, many of which affect the liver.
Correcting Genetic Errors
Hepatocytes, specialized epithelial cells that make up nearly 80% of all liver cells, are key cells that carry out a plethora of biological functions.
Mutations affecting these cells impact function and lead to many common monogenic inborn errors of metabolism. Therefore, correcting these mutations in hepatocytes can potentially be curative for genetic diseases.
Our goal is to treat all monogenic liver diseases using an individualized approach.
We need exceptional and passionate scientists and researchers to help us revolutionize the future of medicine.
Meet Our Strategic Collaborators
In order to advance gene-editing machines, we work hand-in-hand with our strategic collaborators, both in academia and industry, to discover novel genomic science and technologies.
Metagenomi is a precision genetic medicines company that uses its proprietary metagenomics and machine learning platform to discover novel genome editing systems for therapeutic development.
Metagenomi has amassed a large metagenomic database and has the broad capability to mine for novel CRISPR and other effector gene-editing enzymes with unique properties. In 2021, we partnered with Metagenomi to discover and advance new gene-editing systems for in vivo human therapeutic applications.
Life Edit Therapeutics
Life Edit Therapeutics, an ElevateBio company, is a biotechnology company that has built a highly innovative gene-editing platform with one of the world's largest and most diverse collections of novel RNA-guided nucleases (RGNs) and base editors with the potential to make “any edit, anywhere” across the entire spectrum of edits.
Moderna's strategic collaboration, as of 2023, will combine Life Edit's suite of proprietary gene-editing technologies with Moderna's mRNA platform to advance in vivo gene-editing therapies against a select set of therapeutic targets.