Because no parent should ever hear:

We identify rare genetic diseases which are within the reach of current technologies and based on disease biology and clinical manifestation, would be amenable to gene-based therapies. We subsequently design therapeutic strategy and partner with leading scientists around the world to coordinate early-stage lab work and preclinical testing to ensure promising therapies are both effective and safe.
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Once a promising therapeutic strategy is developed (see “Discover & Develop”), Gene2Cure Foundation outlines bespoke translational strategy. We design and organize safety studies, including toxicology, secure clinical-grade manufacturing and manage the preparation of regulatory submissions. We work with medical professionals and hospitals to design clinical trial protocols for Phase I/II studies.

“Time is not our friend” - our main goal is to ensure that promising experimental therapies reach patients as quickly as possible. By navigating global regulatory frameworks, organizing clinical trials or early access programs, and coordinating patient access coordination when needed, we ensure that these life-changing therapies are brought to the patients in the most time and cost-effective manner.
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At all stages of the drug development, Gene2Cure Foundation will take advantage of our established partnerships to secure preferential pricing for external services. We will support families in their fundraising efforts, and we will seek funding opportunities, to make the overall cost of drug development as low as possible, so parents can concentrate on the things that matter the most – the children.
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