Rare Disease Partnerships

We never stop working for people living with rare diseases, providing hope with every breakthrough and every partnership. 
Azam, Gaucher disease

Part of the Rare Disease Community

Our track record in rare disease speaks for itself. For over 30 years, we’ve been discovering and developing therapies that transform the lives of families living with rare diseases.

DNA

Improving Lives Through Partnership

Our partnerships focus on disorders with well-defined mechanisms and high unmet medical need. That’s how we deliver solutions that have a real and meaningful impact on the lives of patients. 

Partnering Success Stories

SOBI

We collaborate with Sobi™ to develop and commercialize treatments for people with hemophilia: ELOCTATE®/Elocta® and ALPROLIX®. We’ve also been jointly developing efanesoctocog alfa, an investigational extended-half-life replacement-factor therapy for hemophilia A.

SIRION

We collaborate with SIRION to develop gene therapies based on tissue-selective adeno-associated virus (AAV) vectors. They’re designed to be efficient, low-dose, and scalable, so we can bring gene therapies to more patients. 

Your partnering leads in rare diseases

For more information, contact our Business development team

Brian Bronk

Brian Bronk

Global Head of Business Development, Neurology, Rare Diseases & Technology Platforms

Guy Griebel

Guy Griebel

Global Head of External Innovation, Rare and Neurological Disorders