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

The FDA grants Orphan Drug designation to Sigilon Therapeutics' (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.

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