Rhythm Therapeutics, Inc. Awarded $3.67 Million NIH SBIR Fast Track Grant to Develop Gene Therapy for Atrial Fibrillation


  • Chicago-based Rhythm Therapeutics is led by founder Dr. Rishi Arora, Professor of Medicine, Clinical Cardiac Electrophysiology at Northwestern University - The Feinberg School of Medicine;

  • Grant is from the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health, and from the Small Business Innovation Research program that can total up to $3.67 million;

  • Atrial fibrillation is the most common heart rhythm disorder, affecting six million people in the U.S. and 33 million worldwide.

Rhythm Therapeutics, Inc. (RTI), a biotechnology company pioneering a revolutionary gene therapy to improve the treatment of atrial fibrillation (AF), was awarded $462,689 from the NHLBI to complete the ‘Phase 1’ segment of development which includes optimizing gene doses and electroporation parameters required for atrial gene delivery.


Upon successful completion of the Phase 1 segment (projected year-end 2021), RTI will be eligible to receive an additional $3,212,600 in NIH funding to carry out the Phase 2 segment, which will include determining optimal dose of gene therapy required to attenuate established electrical remodeling in AF, determining the duration of gene efficacy, and assessing the toxicology profile of targeted genes.


“AF is the most common heart rhythm disorder, affecting six million people in the U.S. and 33 million worldwide,” said Dr. Rishi Arora, CEO of Rhythm Therapeutics and Professor of Medicine, Clinical Cardiac Electrophysiology at Northwestern University - The Feinberg School of Medicine. “Existing standard of care treatments including drugs and ablation have suboptimal efficacy for patients with AF. This is especially the case for patients with more advanced AF or ‘persistent’ AF, which constitutes nearly half of the AF patient population. Improving patient care and avoiding readmissions for high-burden patients is increasingly becoming a focus for payers and healthcare systems.”


Under Dr. Arora’s leadership, Rhythm Therapeutics has identified major molecular mechanisms contributing to AF, identified transgenes to selectively target these mechanisms, and developed a novel method of facilitating gene delivery via electroporation. This has decreased AF in clinically relevant, large animal models of atrial fibrillation.


“The use of targeted electroporation to deliver gene therapy to the heart is an exciting addition to the field of gene therapy,” stated grant principal investigator and gene therapy industry veteran Robert C. Moen, MD, PhD, “and we believe this grant award enables RTI to complete important steps necessary before its promising approach can be tested in the clinic.”


“With support from this valued award, Rhythm Therapeutics has the opportunity to revolutionize the treatment of persistent atrial fibrillation via our proprietary gene therapy platform with the goal of reducing mortality, and improving the quality of life for those suffering with AF worldwide,” said Dr. Arora.


Following projected receipt of the SBIR award on May 1st, 2020, RTI is planning to move company operations to Portal Innovations, a life sciences focused venture development engine located in Chicago's Fulton Market neighborhood providing crafted capital in the form of wet lab & office space, seed investments, managerial expertise and an extensive partner network to promising early stage life science companies developing life-saving technologies for patients.


About Rhythm Therapeutics

Rhythm Therapeutics, Inc., a privately held biotechnology company based in Chicago, is dedicated to delivering the promise of gene therapy for treating serious cardiovascular diseases such as atrial fibrillation. Atrial fibrillation is associated with a 4-5 times higher risk of stroke and heart failure and is a significant health issue worldwide. Its atrial fibrillation program utilizes electroporation to deliver therapeutic transgenes targeting the underlying molecular mechanisms of the disease. Additional information about RTI is available at http:/www.rhythmtherapeutics.com.


About NIH Research Grant

Research reported in this publication was supported by the NHLBI under award number R44HL154912. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NHLBI or the National Institutes of Health.