Rambam Hospital has begun clinical trials of innovative gene therapies targeting transthyretin cardiac amyloidosis (ATTR-CA), a serious and often terminal heart condition caused by protein buildup.

This new therapy employs CRISPR-Cas9 gene editing technology to remove defective genes in the liver, effectively halting the production of harmful proteins responsible for amyloidosis, and offers the potential for a one-time, long-lasting treatment.

ATTR-CA affects approximately 5% of individuals diagnosed with heart failure, leading to symptoms like heart failure, arrhythmias, and increased mortality due to protein accumulation in the heart.

Preclinical trials indicate that the therapy is safe, with no unintended genetic changes, and it involves an intravenous drug administered alongside temporary immunosuppressive therapy.

Administered early, this gene therapy could prevent or significantly slow disease progression, making it especially beneficial for older patients who struggle with complex medication regimens.

The success of this approach may improve early detection of amyloidosis-related heart failure, enabling more effective, personalized interventions.

However, regulatory approval processes in the US, overseen by the FDA’s Center for Biologics Evaluation and Research, remain rigorous, with recent leadership changes potentially impacting the speed and safety of approval.

Leading biotech companies such as Intellia Therapeutics, CRISPR Therapeutics, Beam Therapeutics, and Verve are at the forefront of developing gene-based treatments for non-hereditary diseases, potentially benefiting broader populations.

Beyond amyloidosis, gene editing therapies are being explored for other age-related diseases like high cholesterol, with trials showing promising reductions in 'bad' cholesterol levels without adverse effects.