On August 29, 2026, AskBio announced the presentation of baseline participant characteristics from GenePHIT, one of the largest randomized gene therapy trials conducted in heart failure to date. The Phase 2 clinical trial is evaluating the investigational, one-time gene therapy umiposgene parvec (AB-1002) for its potential to improve cardiac function. The baseline characteristics were presented at the European Society of Cardiology (ESC) Congress.

Umiposgene parvec is an investigational, one-time gene therapy composed of a chimeric cardiotropic AAV2/AAV8 vector capsid (AAV2i8). It is administered directly to the heart to promote the production of a modified version (I-1c) of the naturally occurring protein inhibitor-1, designed to block the action of heart failure-related protein phosphatase 1, thereby improving cardiac contractility by restoring intracellular calcium signaling.

GenePHIT is a randomized, double-blind, placebo-controlled, multicenter Phase 2 trial evaluating the efficacy, safety, and tolerability of umiposgene parvec following a direct cardiac infusion without immunosuppression. Baseline data demonstrate successful recruitment of a representative non-ischemic heart failure with reduced ejection fraction (HFrEF) population receiving guideline-directed medical therapy.

Heart failure is a severe, end-stage manifestation of cardiovascular disease and a major global public health challenge. With over 64 million patients worldwide, a significant unmet clinical need remains. Successful enrollment in the GenePHIT study demonstrates the feasibility of conducting large, multicenter, randomized controlled gene therapy trials in heart failure and provides a critical foundation for evaluating the long-term clinical impact of a single gene therapy intervention.

As part of the broader gene therapy ecosystem, Genevoyager provides end-to-end CRO/CDMO services, from drug discovery to large-scale manufacturing, leveraging both HEK293 and our proprietary Bac/Sf9 AAV production platforms to produce high-quality AAV vectors with minimal impurities and no detectable rcAAV. With 30+ drug development projects and multiple IIT/IND submissions supported, Genevoyager has helped advance AAV-based gene therapies and recombinant protein vaccines into clinical development, including programs targeting Parkinson’s disease, refractory epilepsy, hepatitis B, and cancer, with selected programs having received FDA IND clearance and entered Phase I trials. Backed by extensive experience, we are committed to delivering safe, high-quality solutions to academic, pharmaceutical, and biotech partners worldwide.

Source:

www.askbio.com/baseline-characteristics-phase2-heart-failure-gene-therapy-trial/

www.nature.com/articles/s41591-025-04011-z?utm

Image source:

www.askbio.com/congestive-heart-failure-chf-clinical-trial/