pharmaphorum17d
Why AI and blockchain are the solutions to developing orphan drug
We are in the midst of a significant shift in pharmaceutical drug development, with many leading companies focusing increasingly on orphan drugs targeted at small niche markets. This mammoth-sized ...
Minnesota Daily23d
Families look to ‘orphan drug’ development at U
And at the University’s Center for Orphan Drug Research, researchers are working on multiple new treatments. “To have an orphan product, the condition must be rare and there must be some evidence that ...
Targeted Oncology3d
FDA Grants Orphan Drug Designation to HLX22 for Gastric Cancer Treatment
The FDA granted orphan drug designation to HLX22 for gastric cancer, advancing global phase 3 trials to improve first-line ...
PharmiWeb16d
Global Orphan Drug Market Poised for Significant Growth Amid Rising Demand and Regulatory Support
Orphan drugs are specifically designed to treat rare medical conditions, affecting a small percentage of the population. Governments worldwide are introducing incentives and laws to encourage the ...
Morningstar24d
FDA Grants Orphan Drug Designation to Revolutionary Cystic Fibrosis Therapy
“We are pleased to receive the Orphan Drug Designation from the FDA because of its life-saving impact on the disease and our drug development roadmap,” said Guillermo Marmol, President and CEO ...
KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
KRRO-110 is the first RNA editing development candidate from Korro’s proprietary OPERA TM platform and is currently being evaluated in the Phase 1/2a REWRITE clinical study for AATD. Dosing of the ...
Business Wire16d
ENCell’s EN001 Receives Orphan Drug Designation from the U.S. FDA for Charcot-Marie-Tooth Disease
EN001, a novel therapy under development by ENCell, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Charcot-Marie-Tooth (CMT) disease. Phase 1 clinical trial ...