Cambridge, MA and Durham, NC – February 22, 2023 – Moderna, Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines and Life Edit Therapeutics Inc., an ElevateBio company focused on next-generation gene editing technologies and therapeutics, today announced a strategic research and development collaboration to discover and develop in vivo mRNA gene editing therapies. The partnership will combine Life Edit’s suite of proprietary gene editing technologies, including base editing, with Moderna’s mRNA platform to advance in vivo gene editing therapies against a select set of therapeutic targets. mRNA may hold promise in the delivery of gene-editing technology, which has the potential to treat or cure rare genetic and other diseases.
“This collaboration between Life Edit and Moderna demonstrates the strength of our respective technologies to advance programmable medicines to more specifically target disease,” said Mitchell Finer, Ph.D., Chief Executive Officer, Life Edit Therapeutics and President, R&D, ElevateBio. “Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development. We are excited that partners, such as Moderna, are recognizing the potential of our technology.”
Life Edit’s gene editing platform offers a large and diverse library of base editors and RNA-guided nucleases (RGNs). The RGNs are smaller in size when compared to conventional nucleases, potentially enabling greater versatility for delivery. Life Edit’s nuclease collection features a range of Protospacer Adjacent Motifs (PAMs), short sequences that determine the DNA segments in the genome to which a nuclease can bind. The diversity of Life Edit’s PAM collection enables base editing at more sites than any one nuclease could achieve, offering unprecedented access to the genome to target disease.
“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,” said Eric Huang, Ph.D., General Manager and Chief Scientific Officer, 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.”
This collaboration will apply Life Edit’s diverse collection of novel RNA-guided nucleases (RGNs) and base editors together with Moderna’s mRNA platform with the goal to develop curative therapies for some of the most challenging genetic diseases.
Under the agreement, Life Edit and Moderna will collaborate on research and preclinical studies funded by Moderna. Upon exercising an option for a target, Moderna will assume responsibility for further development, manufacturing, and commercialization. Life Edit will receive an upfront payment and is eligible to receive potential development, regulatory, and commercial milestone payments for each target for which Moderna exercises an option, in addition to tiered royalties on global net sales of products from the collaboration.
In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for both clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna’s capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic.
Moderna’s mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology, and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immune-oncology, rare diseases, cardiovascular diseases, and autoimmune diseases. Moderna Genomics was created to leverage the recent advancement in both the RNA delivery platform and the genomic medicines to create the next generation of in vivo gene editing therapeutics. Moderna has been named a top biopharmaceutical employer by Science for the past eight years. To learn more, visit www.modernatx.com.
Life Edit Therapeutics, an ElevateBio company, is a next-generation genome editing company that has built a highly innovative platform with one of the world’s largest and most diverse collections of novel RNA-guided nucleases (RGNs) and base editors. The platform allows Life Edit to target any genomic sequence and develop novel human therapeutics for the most challenging genetic diseases by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of cell and gene therapies, Life Edit Therapeutics will continue to strengthen its platform of genome-editing enzymes, provide gene-editing expertise to strategic partners, and form other third-party partnerships to discover and develop new therapies.
ElevateBio is a technology-driven company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells (iPSCs), and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. In addition, ElevateBio BaseCamp is a purpose-built, technology-enabled manufacturing platform offering process innovation, process sciences, and current Good Manufacturing Practice (cGMP) manufacturing capabilities for viral vectors, RNA, and cell therapy production. Through BaseCamp, its expanding footprint, and its next-generation enabling technologies, ElevateBio is rapidly growing its collaborations with industry partners while also selectively developing its own portfolio of cellular and genetic medicines. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding:
A strategic research and development collaboration between Life Edit Therapeutics, an ElevateBio company, and Moderna to discover and develop in vivo mRNA gene editing therapies, the terms of that collaboration, and the potential for the collaboration to lead to advances in gene editing technologies as part of Moderna’s broader research and development engine. In some cases, forward-looking statements can be identified by terminology such as "will," "may," "should," "could," "expects," "intends," "plans," "aims," "anticipates," "believes," "estimates," "predicts," "potential," "continue," or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others, those risks and uncertainties described under the heading "Risk Factors" in Moderna's Annual Report on Form 10-K for the fiscal year ended December 31, 2021 and Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2022, each filed with the U.S. Securities and Exchange Commission (SEC), and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date of this press release.
Mary Beth Woodin
Senior Director, R&D Communications
Senior Vice President & Head of Investor Relations
SOURCE: Moderna, Inc.