Sigilon Therapeutics' Fabry Disease Therapy Receives Orphan Drug Designation In US

by Vandana Singh, Benzinga Editor

March 5, 2021 9:23 AM | 1 min read |

The FDA has designated Orphan Drug Status to Sigilon Therapeutics Inc's (NASDAQ: SGTX) SIG-007 to treat Fabry disease.
It is a rare genetic disorder caused by AGAL deficiency and the accumulation of specific substrates within a patient's cells, contributing to multi-organ complications.
SIG-007 comprises genetically modified cells with a non-viral vector to express human alpha-galactosidase A or AGAL.
Among the benefits of Orphan Drug designation in the U.S. is seven years of market exclusivity for the indication, if approved.
Price Action: SGTX shares closed 19.5% lower at $21.86 on Thursday.