News

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
The FDA granted an orphan drug designation to novel hedgehog pathway inhibitor taladegib, also known as ENV-101, for treating ...
Buried within the One Big Beautiful Bill Act is a provision that could improve or even save the lives of the 30 million ...
ImCheck Therapeutics receives EMA Orphan Drug Designation for ICT01 for treatment for acute myeloid leukemia: Marseille, France Thursday, July 24, 2025, 15:00 Hrs [IST] ImCheck Th ...
CAN-2409 is an investigational, off-the-shelf, replication-defective adenovirus engineered to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to tumor cells. When administered with a ...
An Orphan Drug Designation Could Provide Significant Benefits to Regen SAN DIEGO, July 23, 2025 (GLOBE NEWSWIRE) -- Regen BioPharma, Inc. (OTC Pink: RGBP and RGBPP) today provides further insight into ...
Investing.com -- Precision BioSciences (NASDAQ: DTIL) stock rose 5% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its PBGENE-DMD ...
In March 2025, KRRO-110 received orphan drug designation from the U.S. Food and Drug Administration for the treatment of AATD. KRRO-110 is currently being evaluated in the Phase 1/2a REWRITE clinical ...
The U.S. FDA has granted Precision Biosciences Inc.’s PBGENE-DMD orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). PBGENE-DMD uses two complementary Arcus nucleases ...
The U.S. Food & Drug Administration has granted orphan drug designation to Chicago-based Amphix Bio's treatment for acute ...
The FDA designates SH-110 as an orphan drug, offering a safer oral treatment option for glioma patients with swallowing ...