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.
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
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
Global Head of Business Development, Neurology, Rare Diseases & Technology Platforms
Global Head of External Innovation, Rare and Neurological Disorders