Achieved Statistical Significance with Up to 48% Slowing of Clinical Progression on UMSARS Rating Scale - MELBOURNE, Australia and SAN FRANCISCO, Jan. 30, 2025 (GLOBE NEWSWIRE) -- Alterity ...

BOSTON - PepGen Inc. (NASDAQ: PEPG), a biotechnology firm focused on oligonucleotide therapies for severe neuromuscular and neurological diseases, is progressing with its CONNECT clinical program for ...

MB-105, a first-in-class CD5-targeted CAR T-cell therapy, received FDA orphan drug designation for relapsed/refractory T-cell ...

SEED Therapeutics Inc. has announced that its molecular glue therapy, ST-01156, has been granted Rare Pediatric Disease and ...

Seed Therapeutics Inc.’s ST-01156 has been awarded orphan drug designation for the treatment of Ewing sarcoma as well as rare pediatric disease designation by the FDA.

This SPA agreement marks an important milestone in the development of Descartes-08 for MG, providing critical regulatory clarity and a clear path toward potential approval,” said Carsten Brunn, Ph.D., ...

The patent application No. 17/309,952 entitled “An A3 adenosine receptor ligand for use for achieving a fat loss effect”, has been accepted by the US Patent Office, will be issued on February 2 nd and ...

The FDA has approved GRAFAPEX™ and fludarabine as a preparative regimen in acute myeloid leukemia or myelodysplastic syndrome ...

Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ZL-1310, a potential highly active first-in-class ...

Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the ...

Nuvalent, Inc. shares have fallen approximately 30% after promising updates on its two lead NSCLC candidates rallied the ...