Shots:
- Matt spoke about the new research program for multiple genetic segments of arrhythmogenic cardiomyopathy
- He also highlighted Renovacor’s research collaboration with the University of Utah’s Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI)
- The interview shows Renovacor’s pipeline expansion to develop transformative therapies in genetically-driven cardiovascular diseases
Smriti: Please give readers a brief insight into your product pipeline. Also, highlight Renovacor’s plans for the advancement of this product pipeline.
Matt Killeen: We’re developing a pipeline of innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. Our lead program, REN-001, is being developed for BAG3-associated dilated cardiomyopathy (DCM) and uses gene transfer technology to address the monogenic cause of this severe form of heart failure.
In July, we announced the expansion of our pipeline through a research collaboration with the University of Utah’s Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) focused on advancing a novel gene therapy program to treat the three largest genetic segments of arrhythmogenic cardiomyopathy (ACM). Currently, available treatment options do not address the underlying genetics or disease biology of ACM, and patients can continue to experience potentially life-threatening breakthrough events.
Smriti: Discuss your collaboration with the University of Utah’s CVRTI and how this collaboration will work towards accelerating the AAV gene therapy program.
Matt Killeen: Our partnership with the University of Utah’s Nora Eccles Harrison CVRTI is focused on a novel cardiac protein discovered by Robin Shaw, M.D., Ph.D., Professor of Medicine at the University of Utah and Director of the CVRTI, whose team studied its effects in regulating trafficking defects that take place in different forms of heart disease and are considered to be major drivers of disease biology. By seeking to restore expression levels of this novel protein in the heart, we believe that we can circumvent the genetic complexities and diversity seen in different genetic forms of ACM and potentially address multiple genetic segments of this disease with a single therapeutic approach. The program is being developed for the three largest genetic segments of ACM: plakophilin-2 (PKP2), desmoglein-2 (DSG2), and desmoplakin (DSP) associated ACM. The terms of the research agreement grant Renovacor an option for an exclusive license to inventions generated from the collaboration.
Smriti: Please mention the studies being conducted for AAV gene therapy under this collaboration.
Matt Killeen: The collaboration leverages positive proof-of-concept data generated in a genetic mouse model of ACM that was performed by Robin Shaw, M.D., Ph.D., Professor of Medicine at the University of Utah and Director of the CVRTI. We are advancing this AAV-based gene therapy program in parallel for PKP2, DSG2, and DSP ACM and are planning to use a combination of single-cell cardiomyocyte studies in addition to robust in-vivo evaluations in genetic animal models of these different forms of ACM.
Smriti: Shed some light on the key findings obtained from the preclinical studies evaluating a genetic mouse model with Arrhythmogenic-Cardiomyopathy (ACM).
Matt Killeen: Pilot proof-of-concept data have demonstrated that this AAV-gene transfer approach restored gap junction protein trafficking to the intercalated disc and yielded a significant reduction in premature ventricular contractions (PVCs). PVCs are a hallmark of ACM and are considered key triggers of potentially lethal ventricular arrhythmias.
Smriti: Give our readers a brief overview of Renovacor’s plans to proceed with this AAV gene therapy in clinical trials and potential IND applications.
Matt Killeen: While it’s too early to speak to specific IND timelines, in partnering with the University of Utah’s CVRTI, we are seeking to rapidly and efficiently advance this compelling potential therapeutic for these devasting genetic forms of ACM. The expansion of our pipeline with this program further demonstrates our precision medicine approach to developing potentially transformative therapies that target core biological drivers of serious cardiovascular diseases. We look forward to collaborating with the University of Utah and leveraging our expertise in heart muscle biology to develop and advance this novel gene therapy that has the potential to address a significant unmet medical need.
Source: Canva
About the Author:
Matt Killeen is the Chief Scientific Officer at Renovacor. Dr. Killeen has published over 20 peer-reviewed papers on a wide range of topics spanning genetic heart diseases and cardiac electrophysiology and authored a textbook on the role of cardiac electrophysiology in pharmaceutical R&D. Dr. Killeen is a member of the Advisory Council for the Sudden Cardiac Arrest Foundation and has been elected a Fellow of the Royal Society of Biology (FRSB) and a Fellow of the American College of Cardiology (FACC). Dr. Killeen holds a Ph.D. in cardiac electrophysiology from the University of Cambridge and a BSc (Hons) in pharmacology from the University of Leeds.
