Two Massachusetts-based companies, Nanite and Saliogen Therapeutics, are teaming up on a new research project aimed at creating a genetic drug with the potential to treat cystic fibrosis (CF).
“Nanite’s relationship with SalioGen is important in the discovery of safe polymeric nanoparticles for a transformative treatment for CF,” said Sean Kevlahan, PhD, CEO and co-founder of Nanite, in a company press release. These nanoparticles will be used to help deliver gene therapies to cells.
Jason Cole, CEO of Saliogen, added that the new collaboration “represents an important step forward in our mission to accelerate the impact of genetic medicine for patients using our novel gene coding technology.”
Saliogen’s gene encoding technology uses a specially designed enzyme, called a transposase, to insert a new gene into a cell’s existing genetic code. According to the company, the technology is able to precisely insert a new gene of any size into a specific location in a cell’s genome.
Both companies bring proprietary technology to the gene medicine project
In laboratory experiments, Saliogen’s technology has shown the ability to deliver a functional full-size version of the CFTR gene in the genome of human lung cells. CF is caused by mutations in CFTR gene, which provides instructions for making a protein – also called CFTR – that helps regulate mucus production. Mutations in the gene cause the CFTR protein to be missing or malfunctioning, which results in the production of abnormally thick and sticky mucus that triggers most CF symptoms.
By introducing a healthy copy of the gene into lung cells, the gene coding technology could theoretically restore normal mucus production in the lungs of people with CF, regardless of their type of mutation. But delivering this type of genetic medicine to a human’s lung cells is much more complicated than using cells in a dish.
By combining the innovative approaches of both companies, we hope to create a non-viral genetic medicine delivered directly to the lungs and provide the first permanent therapy for people living with CF, regardless of mutation.
This is where the new partnership comes in. Nanite specializes in the development of polymer nanoparticles, which can function as molecular envelopes to deliver gene therapy to cells. Under the agreement between the companies, Nanite will use its proprietary platform, called SAYER, to develop nanoparticles to deliver Saliogen’s technology by providing a healthy copy of CFTR to lung cells.
“By combining the innovative approaches of both companies, we hope to create a non-viral genetic medicine delivered directly to the lungs and provide the first permanent therapy for people living with CF, regardless of mutation,” said Cole.
Kevlahan added that the collaboration “underlines the strength of the SAYER platform’s capacity to design fit-for-purpose vehicles,” noting that Nanite is also involved in research funded by the Cystic Fibrosis Foundation as part of its Pathway to a cure”.
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Image Source : cysticfibrosisnewstoday.com