Cardiac Reprogramming Gene Therapy for Post-Myocardial Infarction Heart Failure
Grant Award Details
Grant Type:
Grant Number:
DISC2-13390
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$1,017,000
Status:
Active
Progress Reports
Reporting Period:
Year 2
Grant Application Details
Application Title:
Cardiac Reprogramming Gene Therapy for Post-Myocardial Infarction Heart Failure
Public Abstract:
Research Objective
The candidate is a gene therapy that delivers cardiac reprogramming factors to convert resident cardiac fibroblasts into functioning cardiac muscle. Thus, it is a regenerative cardiac gene therapy.
Impact
The targeted condition is heart failure arising from myocardial infarction or other insults causing focal heart muscle loss. Cardiac muscle cells are post-mitotic and unable to renew after injury.
Major Proposed Activities
The candidate is a gene therapy that delivers cardiac reprogramming factors to convert resident cardiac fibroblasts into functioning cardiac muscle. Thus, it is a regenerative cardiac gene therapy.
Impact
The targeted condition is heart failure arising from myocardial infarction or other insults causing focal heart muscle loss. Cardiac muscle cells are post-mitotic and unable to renew after injury.
Major Proposed Activities
- Complete test article manufacturing to support pig efficacy study
- Select TN-101 development candidate based on results of pig efficacy study comparing two lead candidates
Statement of Benefit to California:
Heart disease is a leading cause of death in adults and children in California, but there is no current treatment that can promote cardiac regeneration. This research will benefit the state of California by laying the groundwork for development of a one-time treatment that could correct heart disease by generating new heart muscle cells from within the heart. If successful, there is potential economic benefit in terms of productive lives saved and in commercialization of this technology.