A clear R&D strategy and a broad pipeline are essential elements in becoming a leader in the treatment of rare and specialty diseases. With a crisp focus on the patient and caregiver perspective, our pipeline focuses on the development of innovative therapies for patients with unmet needs, in the hope of making a meaningful difference in their lives.
Our R&D strategy is focused on specific therapeutic areas in rare and specialty diseases with unmet medical need, utilizing specific technology platforms. With more than 7,000 known rare diseases, we choose to focus on areas in which we have the experience and talent to efficiently drive programs forward. For example, we have developed core expertise related to enzyme replacement therapies and we complement these capabilities by extending our research into other protein replacement therapies. We also continue to challenge ourselves – exploring opportunities outside of our current focus.
Additionally, we aim to meet the needs of all of our key stakeholders. Patients and their caregivers remain at the center of our thinking, but we also seek to align with regulators and payers, and aim to drive an attractive commercial return to our shareholders.
We foster a culture of innovation and calculated risk-taking. Every program is innovative, but the individual risks associated with programs need to be different so that we have risk balance across our overall pipeline. Our creativity is applied both to our programs, and on establishing creative external partnerships that drive the success of our pipeline. We recognize that we can’t be experts in all technologies, so partnering with the right external experts helps broaden our portfolio.
We aim to ensure consistently high productivity from our pipeline, driven by operational excellence. We recruit outstanding talent and have a clear approach to moving programs through the pipeline. By adhering to this strategy, we are able to continue to rapidly develop innovative therapies for our patients.
Building Our Portfolio
We build our internal research portfolio around high confidence targets in diseases with significant unmet medical need, with an exclusive focus on the area of rare disease.
The core of our research portfolio focuses on diseases where the underlying cause is known. In this way, we begin with an understanding of the disease -- meeting with patients and families to understand their daily struggles, and by characterizing the disease phenotype. Next we look at the underlying biology of the disease and select a molecular target that forms the basis of a potential therapy. Our focus on selecting the best molecular target based on the underlying biology of the disease requires a flexible approach to drug discovery. Internally, our core strength is focused on protein engineering. However, if the best disease target cannot be approached with a protein intervention, we complement our internal efforts through strategic partnerships with world-class technology providers to apply cutting edge solutions to these specific programs. This enables us to discover and develop novel therapeutics across a wide range of rare diseases.
To efficiently progress these novel approaches, we take a hypothesis driven approach with a focus on making critical go/no go decisions throughout each phase of pipeline development. By focusing on specific, disease relevant, targets and by learning everything we can about the disease process along the way, we aim to deliver life-altering therapies for patients with rare diseases.