Regenxbio starts study of gene therapy for rare metabolic disorder

 

• The first participant has been dosed in a 5-subject Phase 1/2 clinical trial evaluating REGENXBIO's (NASDAQ:RGNX) RGX-111 for the treatment of mucopolysaccharidosis type 1 (MPS I), a rare inherited disorder in which the body cannot metabolized large sugar molecules called glycosaminoglycans due to a deficiency in an enzyme called IDUA which leads to organ and tissue damage.
• The estimated primary completion date is November 2021.
• Orphan Drug-, Rare Pediatric Disease- and Fast Track-tagged RGX-111 is a one-time gene therapy designed to deliver the α-l-iduronidase (IDUA) gene directly to the central nervous system (CNS).
• https://seekingalpha.com/news/3640278-regenxbio-commences-study-of-gene-therapy-for-rare-metabolic-disorder