Regado Biosciences develops aptamer-based, antidote-controlled therapeutics designed to give physicians the ability to fine-tune the therapeutic effect for each patient and in each setting.

An aptamer is a single-stranded nucleic acid that can adopt a three-dimensional shape, enabling it to bind tightly to a specific protein target. This ability underscores the potential of aptamers as targeted molecular therapeutics.

Regado’s platform technology, licensed from Duke University, enables the Company to develop antidotes to aptamer-based therapeutics. The design of these antidotes is based upon the aptamer’s sequence and consists of an oligonucleotide complementary to a portion of the aptamer.  When the antidote binds to the aptamer, it causes a conformational change to the aptamer’s three dimensional structure, thereby preventing interaction with its target molecule.

About REG1 - Lead Product Candidate

Regado’s lead product candidate, REG1, is the first specific, direct-acting, antidote-controlled anticoagulant ever described. Regado is developing REG1 for use in patients suffering from acute coronary syndrome who undergo coronary revascularization procedures.  These procedures, which include coronary artery bypass grafting (CABG) and percutaneous coronary intervention (PCI), put patients at high risk for therapy-related bleeding complications.  REG1 is being developed initially to increase therapeutic flexibility and improve patient outcomes in coronary revascularization procedures.

REG1 is a two-component system, consisting of an aptamer-based anticoagulant and its matched antidote.  The REG1 anticoagulant component (RB006) is a single-stranded, nucleic acid aptamer.  RB006 selectively and potently binds to and inhibits Factor IXa, a protein critical to blood coagulation.  The antidote component, RB007, is a complementary nucleic acid that binds to and neutralizes RB006.  The binding of RB007 to RB006 causes the predictable and rapid reversal of the RB006 effect and allows the patient’s blood to return to normal.

This therapeutic flexibility will enable clinicians either to completely reverse or titrate the anticoagulant effect based on patient needs.  For this reason, Regado believes REG1 has the potential to be the anticoagulant therapy of choice from the emergency room to the operating room.