Could gene therapy transform heart failure treatment? - European Medical Journal

Could gene therapy transform heart failure treatment?

Illustration of a heart with a monitor
Words by Isabel O’Brien

A gene therapy for heart failure with preserved ejection fraction (HFpEF), also known as diastolic heart failure, has shown promising early results, raising hopes for millions of patients worldwide.

Medera Inc. presented an update on its Phase 1/2a clinical trial at the 2025 HFpEF Summit, unveiling data on its experimental drug, SRD-002. The therapy aims to address HFpEF at a molecular level by improving the heart’s ability to relax and function efficiently – two key impairments that define the disease.

Heart failure remains a global health crisis, with HFpEF responsible for nearly half of all cases. Unlike heart failure with reduced ejection fraction (HFrEF), where treatments have advanced significantly, HFpEF has long been a therapeutic challenge. Existing drugs, such as SGLT-2 inhibitors, offer symptom relief but do not modify the disease process itself. If approved, SRD-002 could change that.

Early trial results are encouraging. Of the six patients treated so far, four have shown improvements in their New York Heart Association classification at six months, alongside better performance in six-minute walk tests and stabilisation of key heart failure biomarkers. Crucially, no serious adverse events related to the gene therapy have been reported.

The study continues to enrol patients, with hopes that further data will validate SRD-002’s potential as the first disease-modifying therapy for HFpEF. If successful, it could herald a new era for heart failure treatment – one that goes beyond symptom management to addressing the root cause of the disease.

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