The firm, which so far has focused on cancer, expects to file an IND application for a treatment for tyrosinemia type I and alkaptonuria next year.

The FDA has granted orphan drug designation to ligufalimab, a CD47 monoclonal antibody, for treatment of acute myeloid ...

On track for first FDA Biologics License Application (BLA) approved cell therapy in orphan indication relapsed/refractory AL Amyloidosis – Los Angeles, CA, Sept. 18, 2025 (GLOBE NEWSWIRE) -- Immix ...

J & D Pharmaceuticals LLC has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s investigational therapy, JD-004, for the treatment of ...

Grant of First Method of Use Patent Adds New Pillar to Grace’s Multi-layered Intellectual Property and Potential Market Exclusivity Protection PRINCETON, N.J., Sept. 18, 2025 (GLOBE NEWSWIRE) -- Grace ...

Completed patient enrollment within nine months with nearly half enrolled from the U.S.Expect to report Phase 3 topline data in the first half of ...