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.’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.

SEED Therapeutics announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease and Orphan Drug designations to ...

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 ...

Drug discovery is the process through which potential new medicines are identified. It involves a wide range of scientific disciplines, including biology, chemistry and pharmacology. An ...

After Discontinuation of Treatment with QRX003 Complete Reversal of Positive Clinical Improvements Across all Measured Endpoints ...

The goal is to treat familial dysautonomia patients with a single injection.

The NPI-001 (N-acetylcysteine amide) tablets are a proprietary investigational therapy for the treatment of patients with ...

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 ...