Ipsen’s purpose in rare diseases
We are working to uncover the science behind rare diseases. We identify well-defined drug targets to bring medicines that modify the course of rare diseases and alleviate symptoms to improve patients’ daily lives.
We are proud to have delivered extensive indications and medicines in rare liver disease, and to have provided the first approved treatment for an ultra-rare bone disease.
Executing translational science and advancing targeted therapies, our research is focused on the intersection between patient’s lived experiences and innovative science, to deliver meaningful medicines.
How Ipsen drives science with purpose in rare disease
Ipsen’s science in rare cholestatic liver disease
Using science to restore balance
Our focus is on expanding therapeutic possibilities for rare liver diseases where current options remain limited.
We aim to restore bile balance through modulating three different receptors involved in the bile acid system to relieve debilitating symptoms and improve long-term liver health.
IBAT and ASBT inhibitors reduce bile acid reabsorption in the intestine, lowering toxic bile buildup that contributes to pruritus and liver damage. PPAR agonists activate receptors in liver cells to regulate bile acid metabolism, reduce inflammation, and help protect against disease progression.
Our commitment extends beyond symptom relief – we strive to drive breakthroughs that reshape the future of treatment and redefine what’s possible for people with rare liver diseases.
Ipsen’s science in rare kidney disease
Targeting the biology of rare kidney disease
Our science in rare kidney diseases harnesses our deep biological expertise across our therapeutic areas to bring transformational therapies where options are limited.
From precise antibody targeting of BK viral proteins to modulating bile acid balance, we are advancing research in conditions such as BK polyomavirus-associated nephropathy and bile acid–driven kidney injury.
By targeting these mechanisms, we aim to deepen understanding and address the processes that drive kidney damage and dysfunction.
Meet the team
Discover the people driving R&D at Ipsen, advancing science with purpose all the way from discovery to patient impact.
Advancing Science with Purpose
We’re advancing medicines in oncology, rare diseases and neuroscience through science-led innovation. Explore our Neuroscience area to see how we’re transforming innovation into medicine.