Two RTP-Rooted Companies Form Foundational Gene Editing Partnership
By Alex Keown
May 26, 2023
Two companies with deep roots in Research Triangle Park have established a multi-target partnership for the development of gene editing therapies against seven different targets.
Denmark-based Novo Nordisk, which operates three different manufacturing sites in RTP, forged a research and development collaboration with Durham-based Life Edit Therapeutics, a division of ElevateBio. Novo Nordisk will leverage Life Edit’s suite of gene editing technologies to develop new treatments for rare genetic disorders, as well as more prevalent cardiometabolic diseases.
Life Edit Therapeutics has one of the largest arrays of novel RNA-guided nucleases and base editors globally. The company’s next-generation platform provides greater specificity and broader genome access. Life Edit’s gene editing technologies have the potential for any genomic sequence of interest to be removed, added, or altered.
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 technology allows for the targeting of any genomic sequence and develop life-changing therapies for genetic disorders that are considered highly challenging. The platform incorporates a diverse collection of novel RNA-guided nucleases (RGNs) and base editors that provide flexible editing and unprecedented access to the genome. Access to novel RGNs and base editors is essential for increased specificity and broader genome coverage for the development of therapies with Life Edit’s platform.
Earlier this year, Life Edit Therapeutics presented preclinical data from its Huntington’s disease program that demonstrated therapeutically relevant reductions of mutant huntingtin protein following allele-specific editing of the HTT gene in murine disease models. AAV5-delivered Life Edit gene editing systems enabling allele-specific targeting of mutant HTT demonstrated dose-response reductions in striatal mutHTT protein with clinically meaningful reductions of greater than 50% observed. These results support continued evaluation and identification of a development candidate, the company announced at the time.
Life Edit, a 2020 spinout of RTP-based AgBiome Delta and ElevateBio, was fully-acquired by Massachusetts-based ElevateBio in 2021 in order to integrate the company’s genome editing capabilities with its own cell and gene therapies. Life Edit’s genome editing capabilities drew the eyes of Novo Nordisk and its focus on advancing base editing capabilities.
“The advancements we’ve made to our next-generation gene editing platform and base editing capabilities are opening the next frontier of treating disease through DNA editing,” Mitchell Finer, chief executive officer of Life Edit Therapeutics and President, R&D at ElevateBio said in a statement. “Combining our Life Edit platform and the ability to make any edit anywhere with Novo Nordisk’s deep disease biology and engineering expertise will bring us closer to delivering potentially curative therapies for some of the world’s most challenging genetic diseases.”
Base editing is a way to make targeted changes to the sequence of a piece of DNA. It is designed to overcome the limitations of existing approaches and expand the potential of genetic medicine. A method of gene editing, base editing transforms a nucleotide base into another through a process that couples two enzymes, a nuclease and a deaminase to edit the target nucleotide base. It is a precision approach that aims to reduce off-target risks and makes base editing a potentially effective approach for correcting genetic mutations associated with diseases.
Life Edit Therapeutics isn’t the only RTP-based company that uses a base editing approach. Beam Therapeutics, which built an $83 million manufacturing facility in RTP two years ago, has a growing portfolio of base edited assets.
Marcus Schindler, executive vice president and chief scientific officer of Novo Nordisk, expressed excitement about the potential to co-create novel treatments for multiple genetic diseases based on Life Edit’s gene editing technologies. In a brief statement, he said the pharma giant, which has invested in biomanufacturing training programs in RTP, is committed to leveraging technologies that have the chance to create potentially curative treatment options to people living with serious chronic diseases.
Under terms of the collaboration, Life Edit will receive an undisclosed upfront cash payment. The company is also eligible to receive milestone payments of $335 million for the first two programs and up to $250 million each for the remaining five development programs. Once the development programs are identified, Novo Nordisk will be responsible for all research and development costs.
Life Edit will also be eligible for tiered royalties on global sales of the programs should they be approved for commercialization. The deal also provides Life Edit the option to a global profit share on one program. Additionally, Novo Nordisk made an equity investment in Life Edit’s parent company ElevateBio.
Life Edit Therapeutics, which has about 30 employees, is currently growing and will likely continue to grow following the infusion of cash from the Novo Nordisk partnership and ElevateBio’s massive $400 million Series D financing round. The company is hiring for multiple positions, including roles in preclinical development, business development and research.
- About the Author
- Latest Posts
Alex Keown is a freelance journalist who writes about a variety of subjects including the pharma, biotech, and life science industries. Prior to freelancing, Alex has served as a staff writer and editor for several publications.
