New gene therapy for HFrEF passes phase 1 testing

The gene therapy AB-1002 was well-tolerated and associated with positive efficacy outcomes in patients with heart failure with reduced ejection fraction (HFrEF). The promising findings of this phase 1 study led to the development of the phase 2 GenePHIT study, which is currently enrolling patients. “AB-1002 is a cardiotropic investigational adeno-associated viral vector gene therapy, expressing a constitutively active I-1 protein to inhibit protein phosphatase 1 [PP1] activity,” outlined Dr Roger Hajjar (Mass General Brigham, MA, USA) [1]. “The inhibition of PP1 may restore calcium cycling and improve left ventricular function in patients with chronic HF.” The phase 1 NAN-CS101 study tested this treatment in 11 patients with NYHA class III HFrEF. The therapy was well-tolerated, with no treatment-emergent adverse events deemed related to the intervention. “There was 1 death, but this incidence was not attributed to AB-1002 but to substance abuse and ...

Please login to read the full text of the article.

If you have no account yet, please register now.

Posted on 22 May 202517 July 2025