Nanite, Saliogen teaming up to develop genetic medicine for CF
New research project uses Saliogen's novel Gene Coding technology
Two Massachusetts-based companies, Nanite and Saliogen Therapeutics, are teaming up on a new research project that aims to create a genetic medicine with the potential to treat cystic fibrosis (CF).
“Nanite’s relationship with SalioGen is consequential in discovering safe polymeric nanoparticles for a transformative treatment for CF,” Sean Kevlahan, PhD, CEO and co-founder of Nanite, said in a company press release. These nanoparticles would be used to help deliver genetic therapies into 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 Coding technology uses a specially engineered enzyme, called a transposase, to insert a new gene into the existing genetic code of a cell. According to the company, the technology is able to precisely insert a new gene of any size at a specific location in a cell’s genome.
Both companies bring proprietary tech to genetic medicine project
In laboratory experiments, Saliogen’s tech has shown the ability to introduce a full-sized, functional version of the CFTR gene into the genome of human lung cells. CF is caused by mutations in the CFTR gene, which provides instructions for making a protein — also called CFTR — that helps regulate the production of mucus. Mutations in the gene lead the CFTR protein to be absent or dysfunctional, which results in the production of the abnormally thick, sticky mucus that drives most CF symptoms.
By inserting 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 mutation type. But getting this type of genetic medicine to cells in the lungs of a living human being is a lot 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.
That’s where the new partnership comes in. Nanite specializes in developing polymer nanoparticles, which can function like molecular envelopes to deliver genetic therapies into cells. Under the agreement between the companies, Nanite will be using its proprietary platform, dubbed SAYER, to develop nanoparticles to deliver Saliogen’s tech 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,” Cole said.
Kevlahan added that the collaboration “underscores the power of the SAYER platform’s capacity to design fit-for-purpose delivery vehicles,” noting that Nanite also is involved in research funded by the Cystic Fibrosis Foundation as part of its Path to a Cure initiative.
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