The deal will pool together Moderna’s mRNA platform and Life Edit’s gene editing expertise, including an emerging, high-precision form of gene editing called base editing. The aim is to develop treatments or even cures for challenging genetic diseases; details on the specific indications are yet to be disclosed.
Some of the most advanced gene editing therapies involve extracting a patient’s cells, editing their genome in the lab, and returning them to the patient. One prominent example of this so-called ex vivo approach is exa-cel, a therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals in phase 3 testing for the treatment of sickle cell disease. In vivo gene editing therapies are a more recent form of the technology that is delivered directly into the patient. One of the most advanced in vivo gene editing therapies is Intellia Therapeutics’ NTLA-2001, which is in early clinical development for the treatment of transthyretin (ATTR) amyloidosis.
As part of the deal, Moderna and Life Edit will work together on the preclinical development of mRNA-based in vivo gene therapies. Moderna will bankroll this research, and can choose to take a candidate developed in the collaboration into further development and commercialization. In return, Life Edit will receive an undisclosed upfront payment in addition to developmental milestone payments and royalties on any therapies commercialized from the collaboration.
"At Moderna Genomics, we are constantly working to accelerate new therapeutic targets that may one day lead to the next generation of transformative mRNA medicines for patients," stated Eric Huang, general manager and chief scientific officer at Moderna Genomics. "Through our collaboration with Life Edit, we hope to harness the power of gene editing technologies as part of our broader research and development engine, helping to advance our mission and deliver on the promise of mRNA."
mRNA therapeutics are single-stranded ribonucleic acid molecules that allow complex biologic drugs to be produced in the patient’s own body instead of in a traditional factory setting. The technology has been in development for decades, and it proved its potential as a way to deliver vaccines against COVID-19. The rival mRNA giants Moderna and BioNTech in particular gained worldwide recognition for commercializing their COVID-19 vaccine candidates in record time in 2020.
The non-vaccine use of mRNA is also drawing big attention from investors. According to a recent analysis by GlobalData, if five of the leading mRNA therapeutic candidates reach the market, their combined sales could reach over $2 billion by 2028.
One common challenge for in vivo gene editing therapies is their delivery into the patient. For example, the genetic material encoding RNA-guided nucleases – proteins that snip the DNA at a specific location – tend to be larger than many delivery systems can carry. Life Edit Therapeutics, a portfolio company of ElevateBio, gets around this issue by producing RNA-guided nucleases that are smaller than traditional nucleases, making them easier to deliver in vivo.
Other players in the biotech industry are also lining up to combine the potential of RNA with gene editing. In January 2022, Pfizer teamed up with Beam Therapeutics to develop in vivo mRNA base editing therapies for rare genetic diseases of the liver, muscle, and central nervous system. And this week, Brain Biotech inked a deal with Transcode Therapeutics to deliver a precise cancer-busting CRISPR nuclease into tumors using an RNA platform.