Libmeldy (atidarsagene autotemcel)
Metachromatic Leukodystrophy (MLD)
Key Facts
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.
View full company profileAbout 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.
View full company profileOther Metachromatic Leukodystrophy (MLD) Drugs
| Drug | Company | Phase |
|---|---|---|
| HMI-104 / HMI-105 | Homology Medicines | Preclinical |
| Atidarsagene autotemcel | Pfizer | Phase 3 |