Academic research is an important source of promising ideas for new treatments, especially for diseases where few or no treatment options are available. But the step from a discovery in the lab to a treatment for patients is often complex.

Our partner Orfenix is organising a webinar on how researchers can bring their innovation closer to patients in a socially responsible way. During the session, the route from research to patient access will be discussed step by step: from regulatory strategy and clinical development to funding, collaboration, business models and affordability.

The repurposing of guanabenz for Vanishing White Matter disease will be used as a central case study. This case shows how academic research can be translated into patient impact. The project led to GuanaRep, a socially responsible spin-out from Amsterdam UMC in which RARE-NL is involved. Using this case, the webinar will show how scientific, clinical, regulatory and organisational questions come together in practice. Participants will also receive a practical checklist to help assess which development route best fits their own research or innovation.

The webinar is relevant for researchers, clinicians, PhD candidates, patient representatives and others involved in bringing therapeutic research closer to clinical application.

More information and registration: Webinar: how do you build an impact-driven spin-off out of academia? – Orfenix