Crysvita (burosumab)

About Kyowa Hakko Kirin

Kyowa Kirin is a publicly traded, global specialty pharma leader with deep roots in Japanese innovation, strategically focused on high-need therapeutic areas including bone & mineral disorders, hematologic malignancies, and rare diseases. Its success is built on proprietary technology platforms like POTELLIGENT® for enhanced antibody therapeutics and the Orchard Therapeutics acquisition for hematopoietic stem cell gene therapy. The company's strategy combines internal R&D excellence with strategic business development to build a sustainable pipeline of transformative medicines, aiming to deliver its 'Vision 2030' of creating life-changing value for patients worldwide.

About Kyowa Kirin

Kyowa Kirin is a global specialty pharmaceutical leader with a mission to deliver 'life-changing value' by addressing high unmet medical needs through innovative antibody and gene therapies. The company has successfully transitioned into a global player, deriving 72% of its sales from international markets and maintaining a robust pipeline of 18 development programs. Its strategy is anchored in proprietary technology platforms like POTELLIGENT, strategic acquisitions such as Orchard Therapeutics, and a focused therapeutic approach in bone & mineral, hematologic, and rare diseases to drive sustainable growth towards its Vision 2030.

About Ultragenyx Pharmaceutical

Ultragenyx is a mission-driven, commercial-stage biotech dedicated to pioneering novel treatments for serious rare and ultra-rare genetic diseases. Founded in 2010, the company has successfully transitioned to a revenue-generating entity with three approved products and has built one of the largest and most diverse clinical pipelines in the rare disease sector. Its strategy employs a flexible, multi-platform approach—including gene therapy, mRNA, and enzyme replacement—to target the root cause of monogenic disorders. Led by CEO Emil D. Kakkis, M.D., Ph.D., Ultragenyx operates globally with a deep commitment to underserved patient communities.

About Ultragenyx Pharmaceutical

Ultragenyx is a mission-driven, commercial-stage biotech dedicated to pioneering novel treatments for serious rare and ultra-rare genetic diseases. Founded in 2010, the company has successfully transitioned to a revenue-generating entity with three approved products and has built one of the largest and most diverse clinical pipelines in the rare disease sector. Its strategy employs a flexible, multi-platform approach—including gene therapy, mRNA, and enzyme replacement—to target the root cause of monogenic disorders. Led by CEO Emil D. Kakkis, M.D., Ph.D., Ultragenyx operates globally with a deep commitment to underserved patient communities.

Therapeutic Areas