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Carfostin™ Overview Carfostin™ is an intracellular protein therapeutic for the treatment of Chronic Heart Failure (CHF). Carfostin™ is comprised of the delivery of a therapeutic gene, protein Phosphatase-1 Inhibitor-1 (I-1) with the use of Biological NanoParticles (BNPs™) and the Self-Complementary Vector Technology. The biology of I-1 predicts that Carfostin™ will not only improve the contractile function of the failing heart muscle, but will also have beneficial effects in remodeling of the heart in patients with late stage Left Ventricular Dysfunction due to CHF of various etiologies. This novel approach, which represents an important adjunct to existing therapies, will provide a non-invasive treatment for these patients and advance the current standard of care. By combining the BNP™ platform technology and the therapeutic gene (I-1), NanoCor aspires to develop the first viable intracellular protein therapy for treatment of CHF. Relative to existing treatments, NanoCor’s technology allows for four primary benefits to CHF patients: (i) increased heart contractility; (ii) reversal of the remodeling process; (iii) avoidance of arrhythmia; (iv) non-invasive treatment; and (v) long-term, sustained therapeutic benefits. Increased contractility: CHF results in the inability of the heart to pump blood forward at a sufficient rate to meet the demands of the body. In failing human hearts, there is reduced cardiac contractility. Expression of I-1 increases the expression of the gene Phospholamban (PLN) which plays an integral role in cardiac contractility by aiding the movement of Ca2+ during each cycle of cardiac contraction and relaxation, increasing cardiac contractility and cardiac output. In rodent and pig models, expression of I-1 has shown beneficial results in reversing the morbidity and mortality of CHF. Remodeling: Patients with CHF experience a gradual enlargement and weakening of the heart. This process, which occurs in all forms of CHF, is called cardiac remodeling. During cardiac remodeling, the heart gradually changes shape, becoming larger and thinner. Cardiac remodeling is the driving force behind the reduced quality of life experienced by patients with CHF. Based on rodent and pig studies performed to date, I-1 appears to terminate and partially reduce the effects of remodeling on the heart of patients with late stage Left Ventricle Dysfunction due to CHF. Non-invasive Treatment: NanoCor will use proprietary BNP™ technology to deliver I-1 into the heart. The BNPs™ will encapsulate I-1 for delivery into heart cells. BNPs™ are derived from recombinant adeno-associated viruses, which are non-pathogenic viruses naturally existing in the human body. The ability of the BNPs™ to target the heart for delivery of I-1 and the efficient delivery provided by BNPs™ may allow treatment by intravenous delivery. Long-term Therapeutic: BNPs™ are expected to express I-1 over a sustained period. Hemophilic dogs treated with Factor IX which was delivered with AAV have continued to express Factor IX over seven years since they were treated. Humans treated with Factor IX which was delivered with AAV continue to express Factor IX over three years since they were treated. As BNPs™ are derived from AAV, NanoCor expects that patients treated with them will receive sustained therapeutic benefits. Product Pipeline - (Coming Soon)
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870 Martin Luther King Jr. Blvd.
NanoCor Therapeutics, Inc. is engaged in the development of novel therapeutics for the treatment of various cardiovascular diseases at the molecular level. NanoCor’s lead therapeutic is Carfostin™, an intracellular protein therapeutic for the treatment of Chronic Heart Failure (CHF). Carfostin™ is comprised of the delivery of a therapeutic gene, protein Phosphatase-1 Inhibitor-1 (I-1) with the use of Biological NanoParticles (BNPs™) and the Self-Complementary Vector Technology. This novel approach, which represents an important adjunct to existing therapies, will provide a non-invasive treatment for these patients and advance the current standard of care.