JAMA Therapeutics: Pioneering Targeted Gene Therapy for Inherited Heart Disease

Innovation for Health

By: Innovation for Health

3 min read

At the Health~Holland Global Investor Forum 2026, Nazma Ilahibaks, CEO and co-founder of JAMA Therapeutics, presented the company’s approach to developing next-generation gene therapies for inherited heart disease. Watch the video to discover how JAMA Tx is developing proprietary delivery platforms designed to overcome one of the key challenges in cardiac gene therapy: getting therapeutic payloads safely and effectively into the right cells.

JAMA Therapeutics (JAMA Tx) is a preclinical-stage cardiac gene therapy spin-off from the Experimental Cardiology lab at University Medical Center Utrecht in the Netherlands. The company is focused on developing durable, disease-modifying treatments for patients with inherited cardiomyopathies.

Addressing an unmet need in cardiac disease

Inherited cardiomyopathies affect an estimated one in 200 to 500 people. These genetic heart conditions can cause progressive damage to the heart muscle and, in severe cases, lead to heart failure and premature death. Despite advances in understanding the genetic causes of these diseases, treatment remains largely focused on managing symptoms and slowing disease progression. For some patients, heart transplantation remains the ultimate treatment option.

Gene therapy could offer a different approach by addressing the underlying genetic cause of disease. However, delivering therapeutic molecules to the heart remains a significant challenge. The safety, efficiency and targeting limitations of current delivery technologies have constrained the development of cardiac gene therapies.

JAMA Tx aims to address this challenge through proprietary delivery technologies designed specifically to reach difficult-to-access cell types.

Developing new delivery platforms

The company is developing two proprietary cell-derived nanoparticle platforms designed to deliver a range of therapeutic payloads, including RNA, proteins and programmable gene editors.

Rather than relying solely on conventional delivery vehicles, JAMA Tx's approach combines biology-driven targeting with endogenous cargo loading. The goal is to improve the delivery of therapeutic molecules to specific cells while supporting the development of multiple potential therapeutic programmes from the same underlying platform.

This platform approach could potentially extend beyond a single disease or therapeutic modality, allowing the company to explore different types of genetic medicines using its delivery technologies.

From platform technology to therapeutic programmes

JAMA Tx is currently advancing two cardiac gene therapy programmes, with the ambition of developing durable treatments for patients with life-threatening inherited heart diseases.

The company's strategy reflects a broader shift in the gene therapy field: moving beyond simply developing therapeutic genetic payloads to solving the equally important challenge of delivering those payloads to the right place in the body.

For cardiac diseases in particular, effective delivery remains one of the major barriers to translating promising genetic discoveries into treatments. By focusing on delivery technology as a core part of its platform, JAMA Tx aims to create new possibilities for diseases where current therapeutic options remain limited.

Pitching to investors

Ilahibaks presented JAMA Therapeutics' approach to investors and the life sciences community at the Health~Holland Global Investor Forum 2026, highlighting the company's technology, pipeline and potential to address a significant unmet medical need.

The Global Investor Forum connects innovative life sciences companies with investors through company pitches, one-to-one meetings and other opportunities to explore potential partnerships and investment.

JAMA Therapeutics is one of a growing number of Dutch life sciences start-ups developing innovative technologies with the ambition to translate academic research into new treatments for patients.

Watch the video above to hear Nazma Ilahibaks present JAMA Therapeutics and its vision for the future of cardiac gene therapy.

Interested in pitching your company?

Would you like to pitch your company at the next Health~Holland Global Investor Forum and Innovation for Health, taking place on 10–11 March 2027 in Utrecht, the Netherlands? Registration is now open on Global Investor Forum website.


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