FDA Grants Passage Bio Orphan Drug Designation for PBGM01 for Treatment of Infantile GM1 Gangliosidosis
Passage Bio, Inc. (PASG)
Company Research
Source: GlobeNewswire
PHILADELPHIA, April 21, 2020 (GLOBE NEWSWIRE) -- 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 caused by mutations in the GLB1 gene that results in rapidly progressing neurodegeneration. “Infantile GM1 is an incredibly devastating disease that impacts one of our most vulnerable populations,” said Bruce Goldsmith, Ph.D, chief executive officer of Passage Bio. “This designation represents an important recognition of the dire need for an effective treatment option for the children and their families impacted by GM1. We believe PBGM01 has the potential to be life-altering, and we look forward
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News
- The GenScript Biotech Global Forum Makes a Strong Debut in Europe, Highlighting Breakthroughs in Cell and Gene Therapy Development [Yahoo! Finance]Yahoo! Finance
- Passage Bio, Inc. (NASDAQ: PASG) had its "buy" rating re-affirmed by analysts at Chardan Capital. They now have a $7.00 price target on the stock.MarketBeat
- Passage Bio, Inc. (NASDAQ: PASG) had its "buy" rating re-affirmed by analysts at Canaccord Genuity Group Inc.. They now have a $13.00 price target on the stock.MarketBeat
- Passage Bio Achieves Milestones in Gene Therapy Trials [Yahoo! Finance]Yahoo! Finance
- Passage Bio Reports Third Quarter 2024 Financial Results and Provides Recent Business HighlightsGlobeNewswire
PASG
Earnings
- 11/13/24 - Miss
PASG
Sec Filings
- 11/14/24 - Form SCHEDULE
- 11/14/24 - Form SCHEDULE
- 11/14/24 - Form SC
- PASG's page on the SEC website