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Gene Based Therapy for Congestive Heart Failure

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
Program Year/Program:
2012 / SBIR
Agency Tracking Number:
R43HL108581
Solicitation Year:
2012
Solicitation Topic Code:
NHLBI
Solicitation Number:
PA11-096
Small Business Information
NANOCOR THERAPEUTICS, INC.
870 MARTIN LUTHER KING JR BLVD CHAPEL HILL, NC 27514-2600
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2012
Title: Gene Based Therapy for Congestive Heart Failure
Agency: HHS
Contract: 1R43HL108581-01A1
Award Amount: $640,383.00
 

Abstract:

DESCRIPTION (provided by applicant): Congestive heart failure is a major cause of morbidity and mortality in the United States. While progress in conventional treatments is making steady and incremental gains to reduce heart failure mortality, there is a critical need to explore new therapeutic approaches. Gene therapy was initially applied in the clinical setting for inherited monogenic disorders. It is now apparent that gene therapy has broader potential in diseases such as congestive heart failure. Improvement in our understanding of the molecular mechanisms of heart failure, along with the development of novel and safer vectors for gene delivery, have led to the identification of novel targets that are difficult to manipulate pharmacologically but may bemore amenable to gene therapy. In the last few years calcium cycling abnormalities and specifically deficiencies in sarcoplasmic reticulum calcium uptake have been hallmarks of advanced heart failure. The complex of SERCA2a-phospholamban-Protein phospatase 1 has been difficult to target pharmacologically. However the encouraging results from the CUPID trial in which AAV1.SERCA2a gene transfer was found to be safe and demonstrated benefit in clinical outcomes, symptoms, functional status, NT-proBNP and cardiac structure in a phase 2 study, has once again validated calcium cycling as being an important target for heart failure treatment. For this reason, I-1c with its additional benefits is emerging s an important and valid target for the treatment of heart failure. Even though AAV vectors have been shown to be safe in a number of trials including the CUPID trial, they have the following limitations when used in the setting of heart failure: 1) they are not specific for the heart and 2 pre-existence neutralizing antibodies to any individual serotype would result in the exclusion of a large percentage of the patients. In fact in the CUPID trial 50% of the screened heart failure patients had to be excluded because they had neutralizing antibody titers against AAV1. Nanocor Inc. has developed a chimeric of AAV that more specifically targets the heart and escapes the inherent immunity in patients. We have proposed to use of these novel chimeric vectors, which are also known as Bio Nano Particles (BNP), to directlytarget I-1 in experimental models of heart failure. In this phase 1 application, we will carry out a dose-escalation efficacy study of BNP116.CMV.I1c by Intra-Coronary Infusion in a pre-clinical model of heart failure. PUBLIC HEALTH RELEVANCE: While progress in conventional treatments for heart failure is making steady and incremental gains to reduce heart failure mortality, there is a critical need to explore new therapeutic approaches. It is now apparent that gene therapy is a viable option forthe treatment of congestive heart failure. To this end, Nanocor has developed a cardiotropic chimeric of AAV vector targeting the inhibitor 1 protein in cardiomyocytes which it will test in ths phase 1 SBIR grant.

Principal Investigator:

Daniel Sigg
651-955-6502
dcsigg@gmail.com

Business Contact:

Daniel Sigg
651-955-6502
dsigg@nanocorthx.com
Small Business Information at Submission:

NANOCOR THERAPEUTICS, INC.
870 MARTIN LUTHER KING JR BLVD CHAPEL HILL, NC 27514-2600

EIN/Tax ID: 104383321
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No