Moderna Enters Partnership To Advance Gene Editing Therapeutics

Moderna and Metagenomi recently entered into a strategic research and development collaboration to advance new gene editing systems for in vivo human therapeutic applications.

Under the terms of the collaboration, Metagenomi and Moderna will advance a series of in vivo gene editing therapeutics against undisclosed targets.

The companies will utilize Metagenomi’s gene editing tools and leverage Moderna’s mRNA platform and lipid nanoparticle (LNP) delivery technologies to develop curative therapies for patients with serious genetic conditions.

“Metagenomi has demonstrated the power of its proprietary metagenomics approach that mines the Earth’s natural environment to discover next-generation gene editing tools and has developed discovery capabilities with the potential to address multiple diseases,” Eric Huang, PhD, general manager & chief scientific officer of Moderna Genomics (mGx), said in the announcement.

“Their discovery platform and expertise will expand Moderna Genomics’ ongoing efforts to develop innovative in vivo gene editing therapies to address a significant unmet medical need, Huang continued.

Gene editing has the potential to prevent and treat genetic diseases. Most research on genome editing aims to understand diseases using cells and animal models.

Nearly 7,000 rare diseases are affecting about 25 to 30 million Americans. For example, researchers are currently exploring gene editing for cystic fibrosis, hemophilia, sickle cell disease, cancer, heart disease, HIV, and others.

But currently, just two heritable diseases have FDA-approved gene therapies. 

At the end of October, The National Institutes of Health (NIH), FDA, ten pharmaceutical companies, and five non-profit organizations partnered to boost the development of gene therapies for 30 million Americans living with a rare disease. 

Through the newly launched Bespoke Gene Therapy Consortium (BGTC), researchers will examine the steps involved in AAV vector product, vector delivery into cells, and how therapeutic genes are activated in target cells. 

The partnership will provide vital information to improve the efficacy of vector manufacturing and boost the overall therapeutic benefit of AAV gene therapy.