The Foundation

Why do we exist?

It’s a Global Problem, requiring a Global solution

There are estimated 10,000 rare genetic conditions, over 95% of which have no effective treatment.

We are all created equal: Most of the rare genetic conditions occur ”by chance” and affect children irrespective of their geographic location, socioeconomic status, ethnicity, or religion.

While individually rare, these conditions collectively affect millions of children—more than HIV/AIDS and cancer combined. Pharmaceutical companies often overlook these diseases due to high research costs and limited commercial return.

An estimated 5–10% of newborns are born with a rare genetic condition.

Up to 30% of them will not live past age five.

Globally, this equates to 8 million children born each year with a genetic disorder—over 2 million of whom will die due to their condition.

Gene2Cure Foundation is a non-profit organization dedicated to advancing the development and clinical translation of innovative gene therapies for rare and ultra-rare genetic conditions.

Gene2Cure: Giving children with currently incurable diseases a chance at a better life through personalized gene therapies

Gene2Cure is a non-profit organization dedicated to advancing gene therapy for rare and ultra-rare genetic diseases. We aim to transform the future of medicine by focusing on each child individually – ensuring that those who are often overlooked by traditional pharmaceutical programs, receive access to the experimental treatments they urgently need.

Our mission is driven by deep compassion and scientific excellence. Rare and ultra-rare conditions often lack commercial incentive, leaving families with no therapeutic options and no hope. Gene2Cure exists to change that.

The Foundation’s founders – Spela, Leszek and Dusko – bring together uniquely complementary expertise across the entire therapeutic pipeline: from clinical diagnosis, candidate

therapy design, rigorous preclinical safety and efficacy studies, through to clinical-grade manufacturing, regulatory navigation, and clinical trial execution. Our world-class capabilities in gene therapy engineering, translational research, and bespoke clinical trial development form the cornerstone of our work.

To accelerate the time and minimise the cost of bringing innovative therapies to patients, we have built a strong international network of collaborators and scientific advisors. This ecosystem ensures that every project has the highest chance of clinical success and contributes new knowledge to the broader fields of precision medicine and biomedical research – making each subsequent therapy safer and more effective, while minimising the time and costs involved.

We aim to develop cures, not just treatments

Guided by three core principles – discover and develop, translate, and deliver – we strive to provide truly personalized therapies tailored to each child’s genetic profile, regardless of geography, socioeconomic status, ethnicity, or religion. Our treatments aim to correct the underlying genetic cause of the disease, rather than simply alleviating symptoms.

Once a promising therapeutic candidate is identified, Gene2Cure leverages its scientific, clinical, and regulatory expertise – along with strategic partnerships with leading contract research organisations (CROs) and contract development and manufacturing organizations (CDMOs) – to build a customized developmental plan that streamlines the journey from diagnosis to clinical application.

While our primary purpose is to improve the lives of children with rare and ultra-rare diseases, every project we undertake also advances scientific understanding, paving the way for future therapies to be developed more efficiently and at lower cost.

Few people realize that approximately 10% of children born today are affected by one of over 10,000 known genetic conditions. The need for rapid innovation has never been more urgent.

Gene2Cure also engages directly in educational and awareness programs, working together with families and patient organisation to help inform policymakers, healthcare providers, and the public about the unique challenges faced by affected families and the scientific community. Together, we work to accelerate the development of new therapies and change the tragic statistics that define rare diseases today.

At Gene2Cure, we imagine a future where every child has an equal chance at a healthy, fulfilling life – and where a diagnosis of a rare genetic disorder no longer signals the end of hope, but rather the beginning of a well-defined path toward a cure.

DONATE HERE