Research collaboration with the University of
Utah's Nora Eccles Harrison Cardiovascular Research and Training
Institute expands pipeline with the addition of an AAV gene therapy
program for multiple genetic segments of arrhythmogenic
cardiomyopathy
Renovacor, Inc. (NYSE: RCOR), a biotechnology company focused on
delivering innovative precision therapies to improve the lives of
patients and families battling genetically-driven cardiovascular
and mechanistically-related diseases, today announced it has
expanded its pipeline to advance an AAV gene therapy program as a
potential precision therapy for three genetic segments of
arrhythmogenic cardiomyopathy (ACM). To accelerate this new
program, Renovacor has entered into a research collaboration with
the University of Utah’s Nora Eccles Harrison Cardiovascular
Research and Training Institute (CVRTI). The terms of the research
agreement grant Renovacor an option for an exclusive license to
inventions generated from the collaboration.
The research collaboration will focus on a protein discovered by
University of Utah scientists that has the potential to address
multiple genetic segments of ACM. The new program is being
developed as an AAV-based gene therapy to treat potentially
life-threatening arrhythmias associated with the disease by
restoring gap junction protein trafficking and gap junction
communication between heart muscle cells. The program will be
developed for the three largest genetic segments of ACM:
plakophilin-2 (PKP2), desmoglein-2 (DSG2), and desmoplakin (DSP)
associated ACM. Currently available treatment options do not
address the trafficking defects central to each of the these
genetically-driven forms of ACM.
The collaboration leverages positive proof-of-concept data
generated in a genetic mouse model of ACM that was performed by the
Shaw Lab, led by Robin Shaw, M.D., Ph.D., Professor of Medicine at
the University of Utah and Director of the CVRTI. These data
demonstrate restoration of gap junction trafficking to the
intercalated disc and a significant reduction in premature
ventricular contractions (PVCs). PVCs are a hallmark of ACM and key
drivers of potentially lethal ventricular arrhythmias.
“Renovacor’s pipeline expansion with this new AAV gene therapy
research program for multiple genetic segments of ACM further
demonstrates our precision medicine approach to develop potentially
transformative therapies that target core biological drivers of
serious cardiovascular diseases,” said Matt Killeen, Ph.D., Chief
Scientific Officer of Renovacor. “We believe we have found the
ideal program and partner to leverage our expertise in heart muscle
biology to discover and develop a novel gene therapy that could one
day address a significant unmet medical need.”
“We are thrilled to have Renovacor as a partner to continue the
research into these very important genetic drivers of ACM,” said
Robin Shaw, M.D., Ph.D., Director of the CVRTI. “ACM is a serious
disease of heart muscle that can lead to life-threatening,
intractable arrhythmias. The team at Renovacor are experts in the
understanding the importance of heart muscle biology, which makes
them the ideal development partner to advance a novel, precision
medicine approach for ACM. By seeking to understand and address a
key causal disease pathway in ACM, together we hope to develop a
therapeutic that could help improve the lives of patients who are
living with this serious form of cardiomyopathy.”
Arrhythmogenic cardiomyopathy (ACM) is a heritable heart muscle
disorder that can affect the left and right ventricle. It is
characterized by a heightened risk of potentially lethal
ventricular arrhythmias, fibrofatty replacement of myocardial
tissue, and in some patients, heart failure.(1,2) It is recognized
as a disease of the desmosome, with well-defined genetic drivers.
The prevalence of ACM is estimated to range from 1 case in 1,000
persons to 1 case in 5,000, with an average age of diagnosis of
approximately 30 years.(1-3) Current treatment options aim to
prevent potentially life-threatening arrhythmias and progression to
end-stage disease, but they do not target the underlying genetics
or disease biology and, as such, patients can continue to
experience serious breakthrough events.(1-2)
About Renovacor
Renovacor is a biotechnology company focused on delivering
innovative precision therapies to improve the lives of patients and
families battling genetically-driven cardiovascular and
mechanistically-related diseases. The company’s lead program in
BAG3-associated dilated cardiomyopathy (DCM) uses gene transfer
technology to address the monogenic cause of this severe form of
heart failure. Renovacor’s vision is to bring life-changing
therapies to patients living with serious genetic cardiovascular
and related diseases, by developing medicines that target the
underlying cause of disease and provide a transformative benefit
and significant improvement to quality of life.
About the University of Utah
The University of Utah is the state’s flagship institution of
higher education, with 18 schools and colleges, more than 100
undergraduate and 90 graduate degree programs, and an enrollment of
more than 32,000 students. The University serves as a catalyst for
the regional innovation economy, having supported the launch and
growth of over 300 companies and conducted more than $640 million
in annual research.
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1.
Austin KM et al Nat Rev Cardiol. 2019 Sep; 16(9): 519–537
2.
Corrado D, et. al, N Engl J Med 2017;376:61-72
3.
McNally E (2017) in: Adam MP, Mirzaa GM, Pagon RA,
GeneReviews®
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Investors: Brooks Rahmer Renovacor, Inc. 610-424-2627
ir@renovacor.com
Media: Patrick Bursey LifeSci Communications 646-970-4688
pbursey@lifescicomms.com
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