Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy
Grant Award Details
Grant Type:
Grant Number:
CLIN1-16244
Investigator(s):
Award Value:
$4,000,000
Status:
Pre-Active
Grant Application Details
Application Title:
Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy
Public Abstract:
Therapeutic Candidate or Device
RJB-0402 is a liver-targeting adeno-associated virus vector-based gene therapy that drives over expression of FGF21.
Indication
Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening ventricular arrhythmias and sudden cardiac death.
Therapeutic Mechanism
RJB-0402 targets hepatocytes to drive liver specific expression of FGF21 to restore cardiomyocyte function in patients with DSP ACM at high risk of life-threatening arrhythmias and sudden cardiac death.
Unmet Medical Need
Despite treatment with ICDs, antiarrhythmic therapies, and heart failure medications, disease progresses and patients remain symptomatic and at high-risk. There are no disease-modifying treatments for ACM, other than cardiac transplant, for late-stage disease.
Project Objective
RJB-0402 IND Submission to FDA
Major Proposed Activities
RJB-0402 is a liver-targeting adeno-associated virus vector-based gene therapy that drives over expression of FGF21.
Indication
Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening ventricular arrhythmias and sudden cardiac death.
Therapeutic Mechanism
RJB-0402 targets hepatocytes to drive liver specific expression of FGF21 to restore cardiomyocyte function in patients with DSP ACM at high risk of life-threatening arrhythmias and sudden cardiac death.
Unmet Medical Need
Despite treatment with ICDs, antiarrhythmic therapies, and heart failure medications, disease progresses and patients remain symptomatic and at high-risk. There are no disease-modifying treatments for ACM, other than cardiac transplant, for late-stage disease.
Project Objective
RJB-0402 IND Submission to FDA
Major Proposed Activities
- Complete IND enabling nonclinical studies
- Manufacture drug product to support the proposed first-in-human trial
- Complete study startup for a natural history study to support the proposed first-in-human trial
Statement of Benefit to California:
The aim of this project is to complete IND-enabling activities for development of RJB-0402 for the treatment of DSP ACM. We are confident that RJB-0402, a pleiotropic one-time gene therapy, holds great promise as a transformative regenerative medicine, to meet the unmet medical need of DSP ACM patients in California and beyond, as the first disease modifying therapy for this rare disease.