iECURE Receives FDA Orphan Drug Designation for GTP-506, an Investigational Gene Editing Product Candidate for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency
The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States.
- The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States.
- Orphan Drug Designation provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs.
- Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products.
- 1 iECURE has licensed the ARCUS nuclease from Precision BioSciences for four gene insertion programs including OTC, CTLN1 and PKU.