Advancing treatments for rare diseases together

Pierre Fabre and EspeRare move forward a pioneering in-utero therapy for XLHED patients

02Feb

When French pharma company, Laboratoires Pierre Fabre and Swiss not-for-profit organization, EspeRare, joined forces by the end of 2020, their goal was to develop a life-changing treatment for a debilitating rare disease, X-Linked Hypohidrotic Ectodermal Dysplasia, which affects approximately 1 in 25,000 live male births every year.

“Currently, there are no therapeutic options for XLHED. Only the symptoms of the disease can be alleviated” – as Caroline Kant, CEO and co-founder of EspeRare and Caroline Miklaszewski, Project Director at Pierre Fabre, jointly stated.

There is no doubt that the drug development in rare diseases is highly challenging but when two patient-centric organizations combine complementary expertise, the ground-breaking research and a novel therapeutic approach can see a daylight. ER-004, a pioneering in-utero protein replacement therapy, is currently being investigated in the EDELIFE clinical trial and  if approved, it will become the first therapy to address a genetic condition before birth.

To read the full article, please follow the link: Discovery & Development (themedicinemaker.com)