- 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.
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