Research business

Renovacor announces data from a pilot study in pigs showing successful cardiac transduction with REN-001 administered by low-dose retrograde coronary sinus infusion published in the Journal of the American College of Cardiology: Basic to Translational Research

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Renovacor, Inc. (NYSE:RCOR), a biotechnology company focused on providing innovative precision therapies to improve the lives of patients and families struggling with genetically-mediated cardiovascular and mechanistic diseases, announced today the publication of the results of a preclinical study demonstrating cardiac transduction with low doses of REN-001 administered by retrograde coronary sinus infusion (RCSI) in healthy Yucatan pigs. The document, titled “Cardiac Transduction in Mini-Pigs After Low-Dose Retrograde Coronary Sinus Infusion of AAV9-BAG3: A Pilot Study,” was published in the peer-reviewed journal Journal of the American College of Cardiology: from basic science to translational science (JACC: BTS).

REN-001 is an AAV-based gene therapy designed to directly address the underlying cause of SAC3-associated dilated cardiomyopathy (BAG3-DCM) using a validated AAV9 capsid to deliver a functional copy of the SAC3 gene to heart tissue. In the Yucatan pig pilot study presented in JACC: BTSlow doses (SAC3 gene (i.e., vector copy number threshold ≥1). The study also demonstrated diffuse transduction patterns in several regions of the heart and documented the presence of vector mRNA/transcript. In addition, all animals tolerated the procedure without evidence of cardiac injury (eg, arrhythmia, presence of myocardial scar, or coronary sinus injury at necropsy).

The Yucatan pig pilot study published in JACC: BTS included three dose groups. Group A assessed a dose of 1.46e12 vg/kg, Group B assessed a dose of 3.45e12 vg/kg, and Group C assessed a dose of 7.58e12 vg/kg (based on body weight). median of pigs for each group). Levels of vector genomes and corresponding RNA transcripts were quantified. A summary of the data is presented below.


Vector/pig genomes

Cardiomyocyte (mean ± SEM)

Vector Transcripts*

(relative quantities; mean ± SEM)

Group A (n=4)


4.1 ± 1.0

Group B (n=2)

2.0 ± 0.8


Group C (n=1)



*Vector mRNA was assessed using qPCR targeting vector cDNA and expressed as relative amounts (rq) to housekeeping 18S gene

“We believe this publication provides important validation of the RCSI delivery method, which is a key differentiator for our REN-001 program,” said Marc Semigran, MD, Renovacor Chief Medical Officer and co-author of the paper. “By delivering the vector directly to the heart via the coronary venous circulation using a percutaneously placed catheter, we have observed what we believe to be clinically therapeutic levels of cardiomyocyte transduction by our viral vector at doses below those used for the systemic delivery of AAV gene therapies for other genetic diseases. We believe that RCSI delivery of our gene therapy candidate has the potential to provide safety and manufacturing advantages over systemic approaches, which may require higher doses of vector to achieve levels of cardiac transduction similar to those observed in this animal study. We look forward to advancing REN-001 using learnings from the recently published pilot study and are working rapidly towards submitting an IND application expected in the second half of the year. »

Arthur M. Feldman, MD, Ph.D., Founder of Renovacor, Laura H. Carnell Professor of Medicine at Temple University’s Lewis Katz School of Medicine, and the paper’s senior author added, “The levels of transduction achieved with low dose RCSI in this preclinical study are promising and we believe strongly support the advancement of REN-001 into the clinic. More than 80% of BAG3-DCM patients have truncating variants of the SAC3 gene, which reduce levels of functioning SAC3 proteins in the heart. We believe that data showing successful cardiac delivery and transcription of SAC3 in Yucatan pigs provide strong evidence for the ability of REN-001 to potentially correct this genetic defect. Given BAG3-The devastating nature of DCM and the lack of effective therapies represent an important step in addressing a pressing unmet medical need.

This article was published, in its entirety, in the June 2022 issue of JACC: BTS. The article will also be published, in full, in the September 2022 issue of JACC:BTS dedicated to DNA and RNA-based cardiovascular therapeutics.

About Renovacor

Renovacor is a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families struggling with genetically-based cardiovascular and mechanistic diseases. The company’s main program in SAC3-associated dilated cardiomyopathy (DCM) uses gene transfer technology to treat the monogenic cause of this severe form of heart failure. Renovacor’s vision is to bring life-changing therapies to patients with serious cardiovascular and related genetic diseases, by developing drugs that target the underlying cause of the disease and provide transformative benefit and significant improvement in quality of life.

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