Transcription Factor Gene Therapy for Bradyarrhythmias

Introduction

PacingCure has developed BradyTx-01: a novel gene therapy medicinal product (GTMP) to cure life-threatening cardiac pacing dysfunctions by reprogramming cardiomyocytes into pacemaker cells. BradyTx-01 is based on a novel method to optimally control transcription factor overexpression through adeno-associated Virus (AAV) mediated gene transfer. The “biological pacemaker” that is created:

1. Improves cardiac function,
2. Responds to metabolic demand,
3. Can be delivered using minimally invasive techniques, and
4. Potentially lasts a lifetime.

TRACTION Project Overview

In the TRACTION project, PacingCure – together with AUMC – will further optimize AAV-mediated transcription vector gene therapy for pacemaker applications. This will involve validating the novel AAV vector technology in:

• Mice
• Pigs
• Non-human primates

Subsequently, a large-scale manufacturing process will be designed to release the materials that will be used for validating and demonstrating long-term safety and efficacy in porcine studies, thereby establishing preclinical proof-of-concept of BradyTx-01.

Technology-Product-Market Fit

In parallel, we will validate and optimize the technology-product-market fit of BradyTx-01. The evolving value propositions will be tested and optimized with relevant stakeholders. Key activities will include:

• Defining the optimal regulatory strategies
• Performing health technology assessments
• Developing the optimal business strategy

This will set the stage for effective clinical and commercial development of BradyTx-01.

Future Development Steps

Within the TRACTION project, we will take the next steps in our development while also establishing the optimal strategy for our trajectory towards the clinical stage and commercialization of BradyTx-01. The TRACTION project will thus facilitate the complete transition of PacingCure into a fully operational company with an attractive and investment-ready business proposition.