Sigilon Therapeutics' MPS-1 gene therapy an Orphan Drug in U.S.

 

• The FDA grants Orphan Drug designation to Sigilon Therapeutics' (NASDAQ:SGTX) SIG-005 for the treatment of mucopolysaccharidosis type I (MPS-1), 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.
• SIG-005 contains a human cell line genetically modified with a non-viral vector designed to express human a-L-iduronidase (IDUA), an enzyme which is missing or defective in patients with MPS-1. The IDUA enzyme is essential for the breakdown of glycosaminoglycans.
• The company anticipates to initiate a Phase 1/2 trial in this patient population in 2H of 2021.
• The Orphan Drug tag may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the U.S. following marketing approval.
• https://seekingalpha.com/news/3645257-sigilon-therapeutics-mpsminus-1-gene-therapy-orphan-drug-in-u-s