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.
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.
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.