Marina Biotech announced last month that its phase I familial adenomatous polyposis treatment CEQ508 has received orphan drug status from the US Food and Drug Administration.
Separately this month, the company said that it has been issued a notice of allowance from the US Patent and Trademark Office for a patent application covering its so-called DiLA2 delivery technology.
FAP is an inherited, colorectal cancer syndrome characterized by the growth of colorectal polyps. Though the polyps are initially benign, they become malignant in nearly all cases in the absence of colectomy, according to Marina.
CEQ508 is based on Cequent Pharmaceuticals' so-called transkingdom RNAi technology, and is designed to inhibit the oncogene beta-catenin, which is expected to prevent new polyp formation and possibly slow the progression to malignancy of existing ones.
Marina acquired Cequent last year.
"The orphan drug designation provides us with significant regulatory and financial incentives to do everything in our power to rapidly move this compound to market," Marina President and CEO Michael French said in a statement.
Marina expects to dose the first patient in the phase I trial this year.
Meanwhile, the company said it has been notified that the USPTO will grant a patent on an application covering DiLA2 molecules and formulations, pharmaceutical compositions of DiLA2 molecules with therapeutic nucleic acids, and methods for treating human disease using the DiLA2 delivery platform.
The application, No. 20080317839, is entitled, "Amino Acid Lipids and Uses Thereof."