AskBio Advances Heart Failure Gene Therapy in Phase 2

Durham, North Carolina, August 29, 2026

GenePHIT Completes Enrollment Across 12 Countries

The GenePHIT Phase 2 trial successfully randomized more than 170 participants receiving either umiposgene parvec or placebo. The multinational study enrolled patients with non-ischemic cardiomyopathy, NYHA Class III heart failure symptoms and reduced ejection fraction, providing a well-characterized population receiving contemporary guideline-directed medical therapy. Participants were treated across 64 clinical sites in the United States, Canada, the United Kingdom and several European countries. The trial is designed as a randomized, double-blind, placebo-controlled, multicenter study evaluating the efficacy, safety and tolerability of umiposgene parvec following direct infusion into the heart. The enrolled population had substantial cardiovascular disease burden. Nearly half of participants had a history of atrial fibrillation, while almost 45% had an implantable cardioverter defibrillator. Participants continued established heart failure therapies, including beta-blockers, angiotensin receptor-neprilysin inhibitors, sodium-glucose cotransporter 2 inhibitors and mineralocorticoid receptor antagonists. These baseline characteristics are intended to provide an important foundation for interpreting future efficacy and safety findings from the investigational gene therapy.

One-Time Gene Therapy Targets Cardiac Function

Umiposgene parvec (AB-1002) is being evaluated as a one-time gene therapy administered directly to the heart. The treatment is designed to promote production of a modified version, known as I-1c, of the naturally occurring protein inhibitor-1. The modified protein is intended to block the activity of protein phosphatase 1 (PP1), a pathway associated with heart failure, with the goal of improving cardiac contractility by restoring intracellular calcium signaling. GenePHIT administers the investigational therapy through antegrade intracoronary artery infusion using a standard catheter without immune suppression. The clinical study is designed to assess several measures of cardiovascular health, including cardiovascular-related deaths, changes in NYHA classification, left ventricular ejection fraction and six-minute walking distance. The trial’s adaptive design is intended to evaluate whether a single administration of umiposgene parvec can produce meaningful improvements in patients with advanced non-ischemic heart failure.

Safety and Efficacy Data Expected in 2027

Heart failure remains a major global health challenge, affecting an estimated more than 64 million people worldwide. Despite advances in standard therapies, patients can continue to experience progressive ventricular dysfunction, hospitalization and premature death. GenePHIT is therefore investigating whether a gene therapy approach can address underlying cardiac dysfunction and potentially provide a longer-lasting therapeutic effect following a single administration. AskBio expects the first efficacy and safety outcomes from GenePHIT in the first half of 2027. These results will be important for determining the continued clinical development of umiposgene parvec in heart failure. The company has emphasized that the successful recruitment of a large multinational randomized gene therapy study demonstrates the feasibility of evaluating this therapeutic approach across diverse clinical settings. Importantly, umiposgene parvec has not been approved by any regulatory authority, and its safety and efficacy have not been established. The upcoming clinical results will therefore represent an important milestone in assessing the investigational therapy’s potential in patients with heart failure.

Source: AskBio press relese