ANTISENSE OLIGONUCLEOTIDE THERAPIES
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 infrastructure needed to develop promising therapeutic approaches and advance them toward patients.
Most ultra-rare diseases have no approved treatment
For people living with ultra-rare genetic diseases, treatment options are often limited or nonexistent. Advances in genetic medicine have made it possible to develop therapies for increasingly small patient populations, but traditional drug development models are rarely suited to diseases affecting only a handful of people.
WinQure is building a different approach, designed to make the development of therapies for ultra-rare diseases scientifically and practically possible.
>6K
distinct ultra-rare diseases, each affecting fewer than 1 in 50,000 people
~100M
people worldwide are currently living with an ultra-rare disease
>99%
of ultra-rare diseases have no approved treatment
OUR PIPELINE
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 changes they target and the therapeutic approaches we are pursuing.
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.
