At Orfenix, our projects reflect our dedication to public-private partnerships and making effective treatments widely available. We repurpose existing compounds to unlock new therapeutic potentials, delivering innovative treatments efficiently and affordably.
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Explore our portfolio.
Our collaborative approach brings together leading academics, commercial partners, and stakeholders to develop sustainable healthcare solutions. With a focus on socially responsible licensing, we ensure our advancements are accessible to patients around the world. Explore our portfolio to see how Orfenix is transforming healthcare through innovation and collaboration.
Orfenix initiated the development of an reformulated cysteamine by bringing key partners together in order to find an alternative development pathway in drug repurposing. Orfenix provides the clinical and regulatory support for Patient One and its mission to develop a product that is effectively available to patients suffering from cystinosis, a rare disorder that often appears in very young children and causes kidney failure, vomiting, and can affect patients’ eyes.
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Orfenix co-founded and manages Haltex Therapeutics, in addition it provides regulatory and clinical expertise to develop the repurposed etidronate as a drug for the treatment of a rare blood vessel disease, PXE. PXE causes patients to lose eye-sight, develop a waxy yellow skin, and suffer from bleeding in the gut.
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Orfenix provides protocol and regulatory support to Guanarep to develop a treatment for a rare but debilitating neurological disease that affects children from a young age and for which there is currently no treatment. The disease leads to severe brain damage, and eventually death.
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