FDA
Fujirebio Diagnostic's Alzheimer's Blood Test Receives FDA 501(k) Clearance
The test, which aids in the detection of the brain amyloid pathology characteristic of Alzheimer's, is the first blood test cleared by FDA for use in diagnosing the disease.
At ASGCT, Researchers Share Plans to Administer Gene Therapy for Lysosomal Storage Disease to Fetus
Premium
The treatment has been used in a trial to treat children with GM1 gangliosidosis, but if the FDA allows, this would be the first time a gene therapy is given to a fetus.
FDA Grants Regenxbio Priority Review for MPS II Gene Therapy Regulatory Application
The FDA will issue a decision on the BLA for RGX-121 by November, which if positive, could make it the first gene therapy for Hunter syndrome.
Hoth Therapeutics Eyeing Human Trials for HT-KIT Antisense Therapy in Early 2026
After seeing promising results in preclinical studies, the firm hopes to begin Phase I trials shortly after getting IND clearance.
FDA Clears Ensoma's IND to Launch Immune Disorder Gene Therapy Trial
EN-374 is a gene therapy designed to treat X-linked chronic granulomatous disease by modifying hematopoietic stem cells in vivo.
Apr 1, 2025