Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy

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Grant Award Details

Grant Number:
CLIN1-16244
Investigator(s):
Institution:
Type:
PI

Disease Focus:
Award Value:
$4,000,000
Status:
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

  • 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.