Copernicus Therapeutics Inc.

Nucleic Acid Nanoparticles for Treating Human Diseases



Copernicus Therapeutics, Inc. is a clinical stage company and a leader in the emerging field of “nano” nucleic acid therapeutics and delivery systems.  The Company’s proprietary platform technology enables new classes of therapeutic agents for the safe and efficient delivery of DNA, RNA, and shRNA. 

Copernicus' nucleic acid nanotechnology has been validated for several diseases in different tissue types, including the development of a first-in-class therapeutic product for the treatment of Cystic Fibrosis (CF).  Results of Copernicus' initial proof-of-concept Phase Ib/IIa dose escalation clinical trial in subjects with cystic fibrosis demonstrated that DNA nanoparticles (NPs) efficiently transferred their gene payload to the nuclei of differentiated airway cells without generating toxicities or inflammation.  The results of the clinical trial have been published after review by scientific peers in the field. 

Copernicus' platform technology supports the development of additional first-in-class therapies to treat other serious diseases with few therapeutic options, including ocular (eye) disorders such as macular degeneration, diabetic retinopathy, glaucoma, and retinitis pigmentosa (RP); neurological (brain) disorders such as stroke, Parkinson’s disease (PD), multiple sclerosis, traumatic brain injury, and Alzheimer’s disease; and pulmonary (lung) disorders other than cystic fibrosis, such as chronic obstructive pulmonary disease, asthma, and viral lung infections such as influenza or SARS. 

Copernicus has focused its initial efforts on the lung manifestations of cystic fibrosis, with earlier programs in Parkinson’s disease and retinitis pigmentosa to demonstrate the ability of our technology to treat diseases in these different tissue targets.  Each of these tissues have cells that express a certain cell surface protein (nucleolin) that binds tightly to our DNA NPs.  Binding to nucleolin results in the cell taking up our NPs and rapidly delivering them to the nucleus of the cell, where they can be used to express a therapeutic protein.  This use of the patient’s own cells to manufacture the therapeutic protein permits treatment of the diseases while reducing off-target toxicities.  Moreover, our DNA NPs can produce intermediate or long term expression of the therapy, based on the clinical needs of a given indication.