Can Gene Therapy be the Solution for Congestive Heart Failure?


Congestive heart failure accounts for half a million deaths per year in the United States. Despite its place among the leading causes of morbidity, pharmacological and mechanic remedies have only been able to slow the progression of the disease. Today's science has yet to provide a cure, and there are few therapeutic modalities available for patients with advanced heart failure. There is a critical need to explore new therapeutic approaches in heart failure, and gene therapy is emerging as a possible viable alternative. Research has suggested that protein phosphatase inhibitor 1 (I-1c) is down-regulated in failing hearts. AskBio is conducting clinical research to determine if gene therapy to express an active form of I-1c may improve heart function and provide a new approach to treating CHF. The webinar will briefly go over CHF, what is cardiac gene therapy, an overview of how AskBio’s NAN-101 may address the problem and information on the upcoming phase II GenePHIT trial.  


The content in this webinar is created independently by the sponsor. The views and/or opinions expressed in this forum do not purport to reflect the opinions or views of the HFSA or its members.

The activity Can Gene Therapy be the Solution for CHF? is not sponsored or endorsed by the HFSA.

The webinar is not offered in the HFSA-accredited continuing education catalog; therefore, it is ineligible for continuing education units. No committee members or representatives of the HFSA have been involved in the planning of this activity

Learning Objectives:


  • Provide an overview of cardiac gene therapy in congestive heart failure
  • Discuss the mechanism of action of the investigational therapeutic gene therapy NAN-101, which induces the constitutive expression of I-1c
  • Review preliminary data from the phase I trial of NAN-101 in congestive heart failure 
  • Preview the study design for the upcoming phase II GenePHIT trial in congestive heart failure



  • Alain Lamontagne, PhD
  • Roger Hajjar, MD
  • Litsa Kranias, PhD
  • Tim Henry, MD, MSCAI


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