The Cystic Fibrosis Foundation (CFF) is awarding Life Edit Therapeutics up to $400,000 to explore its new gene-editing technology to develop treatments for certain patients with cystic fibrosis (CF) who cannot benefit from existing medicines.
“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,” Mitchell Finer, PhD, CEO of Life Edit, said in a press release.
According to the CFF, Life Edit is seeking to improve base editing, a variation of more traditional gene-editing methods that may make it easier to deliver components to cells.
“By funding this award, we are taking an early step along this long journey to developing gene editing as a potential therapy for people with cystic fibrosis,” William Skach, MD, executive vice president and chief scientific officer of the CFF, said in a separate press release.
CF is caused by mutations in the CFTR gene that result in the production of a faulty CFTR protein. One type of CF-causing mutation, known as a nonsense mutation, creates an early stop signal in the gene sequence, resulting in the production of a shorter CFTR protein that doesn’t work properly. About 13% of all CF cases have nonsense mutations.
Despite increasing advances in therapeutic options for CF, including those targeting common CFTR mutations, nearly 7% of patients with the inheritable progressive disease cannot be treated with available options, Finer said.
With this new funding, the CFF will provide Life Edit with materials, resources, and expertise. This will enable the company to screen its library of gene-editing tools for options that may correct the six most common nonsense mutations in CF patients. Collectively, gene editing allows genetic material to be removed, added, or altered at a specific location in the genome.
“Gene editing holds promise as a way to permanently correct mutations in the cell and — hopefully, one day — provide a cure,” Skach said.
In particular, Life Edit is working to explore its collection of adenine base editors, or A-base editors. Due to their small size, these base editors allow for an easier delivery of gene-editing components in adeno-associated virus vectors — a modified harmless form of a virus — to specific cells in the lungs. Notably, adenine is one of four chemical bases in DNA.
The editors and the specific enzymes used in the technology are developed using harmless microbes, according to Life Edit. That boosts the fidelity and functionality of gene-editing tools while lowering the risk of immune reaction.
“We believe our base editor technology has potential to make a great impact in the lives of cystic fibrosis patients,” said Allie Crawley, PhD, the project’s principal investigator and a member of the Life Edit team.
This support is part of the Foundation’s initiative Path to a Cure — which has a goal of addressing the root cause of CF. The $500 million Path initiative was launched in October 2019.
“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,” Crawley added.
Finer said Life Edit is hoping its gene-editing technology also can be used for other disorders.
“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,” Finer said.
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