CF Foundation Invests in SalioGen’s Gene Coding Therapy

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The Cystic Fibrosis Foundation has announced an investment in SalioGen Therapeutics to support the company’s preclinical research toward  developing a new gene therapy for cystic fibrosis (CF).

SalioGen is working to advance a novel type of gene therapy, called Gene Coding, which is accomplished using the company’s Exact DNA Integration Technology, or EDIT. This technology aims to insert new pieces of DNA into specific places in a cell’s genetic code, which could be exploited to turn genes on or off, or modify their function.

The company’s technology takes advantage of genome engineering enzymes called Saliogase. According to SalioGen, Saliogase “seamlessly inserts new DNA of any size into precise, defined genomic locations.”

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While SalioGen had been exploring this novel technology in other genetic diseases, the new investment will facilitate testing the approach in cystic fibrosis. CF is caused by mutations in the gene CFTR, leading to defects in the CFTR protein, which results in the abnormally thick mucus and other symptoms that characterize the disease.

“SalioGen’s Gene Coding approach seeks to insert a large piece of healthy CFTR DNA at a precise location within the CFTR gene, which could enable the expression of a functional CFTR protein in essentially all individuals with CF,” Martin Mense, PhD, said in a press release. Mense is CF Foundation senior vice president of drug discovery and director of the CF Foundation Therapeutics Lab.

Mense added that such an approach is expected to be effective regardless of the specific mutation(s) in the CFTR gene found in a given patient. By contrast, other therapies like CFTR modulators are effective only in people with specific mutations.

The new investment is part of the CF Foundation’s “Path to a Cure,” a $500 million initiative that aims to speed up the development of experimental treatments that aim to address the root cause of CF — whether through gene therapies, or other methods that restore functionality of the CFTR protein. Details of the investment were not disclosed.

“Genetic therapies are the key to curing CF and, while it is still very early days in this scientific quest, we’re investing in many different approaches that offer promise,” Mense said.