From ultra-rare diagnosis to therapeutic opportunity
WinQure Medicine develops precision antisense oligonucleotide (ASO) therapies for people living with ultra-rare genetic diseases. We combine scientific expertise with the development infrastructure needed to advance promising therapies from academic research toward clinical use.
Most ultra-rare diseases have no approved treatment
For people living with ultra-rare genetic diseases, treatment options are often limited or non-existent. Today, patient foundations, patient organisations and families work with academic researchers to develop therapies targeting specific genetic variants and their associated, often smaller, patient populations. Despite significant scientific advances, many promising therapies remain in academia because traditional drug development models focus on markets with larger patient populations.
WinQure Medicine brings together scientific expertise, patients and development infrastructure to advance these therapies from academic research toward clinical use.
Make your need for treatment visible
RAREkey is our free patient registry, giving people with ultra-rare genetic diseases a way to make their need for treatment known to those who could develop one.
It also creates an opportunity for patients and families to provide input into future therapy development.
Active programs
We are developing ASO therapies for a growing number of ultra-rare genetic diseases, with programs at different stages of development.
Explore our active programs, the genetic variants they target and the therapeutic approaches we are pursuing.
