Renovacor, Inc. announced the publication of the results of a preclinical study demonstrating cardiac transduction with low-doses of REN-001 delivered via retrograde coronary sinus infusion (RCSI) in healthy Yucatan pigs. REN-001 is an AAV-based gene therapy designed to directly address the underlying cause of BAG3-associated dilated cardiomyopathy (BAG3-DCM) by using a validated AAV9 capsid to deliver a functional copy of the BAG3 gene to cardiac tissue. In the pilot Yucatan pig study featured in JACC: BTS, low doses (<1e13 vector genome (vg) per kilogram (kg)) of REN-001 delivered locally to the heart using RCSI resulted in each evaluated cardiomyocyte containing, on average, at least one copy of the delivered BAG3 gene (i.e., vector copy number threshold =1).

The study also demonstrated diffuse transduction patterns across multiple regions of the heart and documented the presence of vector mRNA /transcript. Additionally, all animals tolerated the procedure without evidence of heart injury (e.g., arrythmia, presence of myocardial scar, or coronary sinus injury at necropsy). The pilot Yucatan pig study published in JACC: BTS included three dose groups.

Group A evaluated a 1.46e12 vg/kg dose, Group B evaluated a 3.45e12 vg/kg dose and Group C evaluated a 7.58e12 vg/kg dose (based on median pig weights for each group). Levels of vector genomes and corresponding RNA transcripts were quantified.