Life Edit receives $400K to find gene editing approaches for cystic fibrosis

By The Science Advisory Board staff writers

March 24, 2021 -- Life Edit Therapeutics has received a $400,000 award from the Cystic Fibrosis Foundation to identify potential gene editing approaches to treat patients with cystic fibrosis.

The company will use the funds to screen its library of proprietary base editors for a potential treatment targeting people with cystic fibrosis who are not able to be treated with small-molecule treatments due to nonsense genetic mutations.

Approximately 13% of people living with cystic fibrosis 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 a shortened, nonfunctional protein.

Life Edit will explore its large collection of adenine base editors (A-base editors) that can potentially be used to correct the six most common class 1 cystic fibrosis nonsense mutations to restore CFTR function in vivo. The company's base editors are small in size, allowing in vivo delivery with adeno-associated virus (AAV) vectors to specific tissue types in the lung.

As part of the agreement, Life Edit will benefit from materials, resources, and expertise from the Cystic Fibrosis Foundation. The 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 cystic fibrosis.


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