JR-171 (lepunafusp alfa)
Fabry Disease
Phase 3Active
Key Facts
About JCR Pharmaceuticals
JCR Pharmaceuticals has established itself as a premier global specialty pharmaceutical company focused on high-unmet-need rare diseases, particularly lysosomal storage disorders. Its strategic advantage is anchored in the proprietary J-Brain Cargo® platform, which enables targeted delivery of biologics across the blood-brain barrier—a key differentiator in treating neurological manifestations. The company has successfully commercialized products in multiple international markets and maintains a robust clinical pipeline, driving its significant market valuation. JCR's fully integrated model, from R&D to global commercialization, combined with a strong venture spirit, positions it for sustained growth in the orphan drug space.
View full company profileOther Fabry Disease Drugs
| Drug | Company | Phase |
|---|---|---|
| Corrector Program | Octant Biotech | Hit to Lead |
| GMAC for Fabry Disease | CellGenTech | Preclinical |
| Undisclosed Gene Therapy | uniQure | Preclinical |
| Lucerastat | Idorsia | Phase 3 |
| PRX–115 (pegunigalsidase alfa) | Protalix BioTherapeutics | Submitted |
| Isaralgagene civaparvovec (ST-920) | Sangamo Therapeutics | Phase 1/2 |