- Company to use its novel gene editing technology to explore a potential in vivo gene therapy treatment for cystic fibrosis
Research Triangle Park, NC – March 23, 2020 – Life Edit Therapeutics Inc, a next generation gene-editing company, today announced that it has received an award from the Cystic Fibrosis Foundation to identify potential gene editing approaches to treat certain patients with cystic fibrosis (CF). The award will enable LifeEdit to screen its library of proprietary base editors for a potential treatment targeting people with CF that are not able to be treated by existing small molecule treatments due to what are known as nonsense genetic mutations (also known as “stop” mutations). This award is part of the Cystic Fibrosis Foundation’s Path to a Cure initiative that was launched in October 2019 to address and treat the underlying cause of CF.
“Due in large part to the efforts of the CF Foundation’s support for the development of new medicines, there are now effective therapies available to most people living with the disease, but there remain as many as 7% of patients with CF for whom recent medical advances are not effective,” said Mitchell Finer, Ph.D., ChiefExecutive Officer, Life Edit Therapeutics. “We’re looking forward to working with the CF Foundation to leverage the unique benefits that our platform offers to develop a highly targeted gene editing approach for these individuals. We believe our science and this approach can be applied across a range of diseases, which will be our focus as we work to build a pipeline of life-changing therapies for severe genetic diseases like CF.”
Dr. Allie Crawley, Principal Investigator for the project and member of the Life Edit team, continued by saying, “We are thankful to be a part of the Path to a Cure initiative from the CF Foundation which is focused on curing cystic fibrosis by addressing the underlying cause of the disease. We believe our base editor technology has potential to make a great impact in the lives of cystic fibrosis patients with nonsense mutations and are excited about the opportunity to begin early research in this development.”
Despite tremendous progress in advancing therapeutics to help people with CF live longer and healthier lives, there remain unmet needs to help all those living with this disease. Approximately 13% of people living with CF have nonsense mutations. These mutations cause the cells to stop the production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein midway through the process, resulting in shortened, non-functional protein.
As part of the $400,000 award from the Foundation, Life Edit will explore its large collection of adenine base editors, or A-base editors, that can potentially be used to correct the six most common, Class I, cystic fibrosis nonsense mutations to restore CFTR function in vivo. A unique feature of the base editors under development by Life Edit is their small size which will allow in vivo delivery with Adeno-associated viruses (AAV) vectors to specific tissue types in the lungs. As part of the agreement, Life Edit will benefit from materials, resources, and expertise from the Cystic Fibrosis Foundation.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare genetic disease found in more than 30,000 people in the U.S. CF is a hereditary disease that affects the lungs and digestive system that is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that affect the production of the CFTR protein. When the CFTR protein is not made correctly, it affects the balance of salt and fluids inside and outside of the cell. This imbalance leads to thick, sticky mucus in the lungs, pancreas, and other organs. In the lungs, the mucus clogs the airways and traps germs, like bacteria, leading to infections, inflammation, respiratory failure, and other complications. CF is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.
About Genome Editing and Life Edit Therapeutic’s Platform
Genome editing technologies have revolutionized the way cell and gene therapies and regenerative medicines are discovered and developed by allowing genetic material to be removed, added, or altered at specific locations in the genome.While these technologies are in widespread use experimentally, enzymes that offer broader coverage and greater specificity are needed for creating novel cell and gene therapies.
To meet the need for better genome editing approaches, Life Edit Therapeutics has built one of the world’s largest and most diverse arrays of novel RNA-guided nucleases (RGNs) and base editors that are active in mammalian cells. TheseRGNs were developed using AgBiome’s proprietary collection of more than 90,000microbes and their complete genomes. Life Edit Therapeutics is investigating these proprietary RGNs, which are sourced exclusively from non-pathogenic organisms, to develop new gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. Life Edit Therapeutics’ nuclease collection also has abroad range of Protospacer Adjacent Motifs (PAMs) – short sequences that must follow the targeted DNA sequence in order for the enzyme to make cuts – that offer unprecedented access to genomic loci of interest. The Life EditTherapeutics’ RGNs offer flexible editing options which encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with its proprietary deaminases.
Life Edit Therapeutics’ next generation editing systems will propel the development of novel human therapeutics 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 gene therapies, Life Edit Therapeutics will continue to build its platform of novel nucleases, provide gene editing expertise to strategic partners and ElevateBio’s portfolio companies, and form other third-party partnerships to discover and develop new therapies.
About Life Edit Therapeutics Inc.
Life Edit Therapeutics is a next-generation gene editing company that has built a highly innovative genome editing platform with one of the world’s largest and most diverse collections of novel RNA-guided nucleases (RGNs) and base editors. Life Edit Therapeutics’ next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. The company is continuing to strengthen the platform, developing a pipeline of in vivo gene therapies to address severe genetic disease, and sharing its expertise through strategic partnership. Life Edit is an ElevateBio portfolio company. For more information visit lifeeditinc.com
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