Azam, Gaucher diseasePart 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.
DNAImproving 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
Global Head of Business Development, Neurology, Rare Diseases & Technology Platforms
Guy Griebel
Global Head of External Innovation, Rare and Neurological Disorders