Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead product candidate, PBGM01, for the treatment of infantile GM1 gangliosidosis (GM1). GM1 is a rare and often life-threatening monogenic recessive lysosomal storage disease ca
April 21, 2020
· 6 min read