Genetic Disease


Orchard Therapeutics Granted FDA Orphan Drug Designation for OTL-102 for X-linked Chronic Granulomatous Disease (X-CGD)

Orchard Therapeutics announced the U.S. FDA has granted Orphan Drug Designation for OTL-102, the company’s ex vivo autologous hematopoietic stem cell (HSC) gene therapy being investigated for the treatment of X-linked chronic granulomatous disease (X-CGD). The FDA may...

Hong Kong Baptist University Researchers Achieve Preclinical Breakthrough for Osteogenesis Imperfecta Treatment & Receive FDA Orphan Drug Designation

Researchers from the School of Chinese Medicine at Hong Kong Baptist University (HKBU) have successfully developed a novel aptamer for the treatment of osteogenesis imperfecta (OI) with the aid of artificial intelligence technology. This represents the first time that...

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