To advance research and ensure the best care for patients, we believe having close partnerships with healthcare professionals (HCPs) is key. We therefore welcome you to reach out to us with any query or if you are interested in trial participation email us directly at firstname.lastname@example.org.
Healthcare professionals Inherited retinal disease resources
Our mission is to help people living with rare genetic diseases that currently have no treatment options. We focus on developing intravitreal RNA therapies that treat the underlying cause of inherited retinal diseases (IRDs).
RNA therapy – a fundamentally new approach to IRDs
For the vast majority of the more than 2 million people living with an IRD, there is no treatment available. We are working hard to change that. We are developing several RNA therapies that could potentially stop disease progression or reverse vision loss in currently untreatable diseases.
What is RNA therapy?
RNA therapy is different from gene therapy and gene editing because it doesn't alter DNA. RNA therapy aims to repair RNA, which is a product of DNA, therefore any changes made to the RNA are reversible. The routine intravitreal (IVT) injection of RNA therapies and the potential to treat the whole retina, make it an innovative and accessible approach with a manageable benefit/risk profile.
Our broad pipeline of investigational RNA therapies is evolving, and we are currently conducting several clinical trials. Find below the clinical trials for which we are recruiting patients.Our clinical trials
Please contact us if you are interested in scheduling a (virtual) meeting during the upcoming congresses.View events
Our passion for patients and to reverse blindness drives us to constantly innovate. As such, we work and collaborate with experts in the field to bring our research findings to the world.Access publications
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Expert perspective's series
This video series with expert physicians and patients providing an in-depth review of topics related to our clinical trials data, pipeline and platform to inform the medical community.See the series