drug · gene
rebisufligene etisparvovec
pre_registrationupdated 5mo agoby $RARE Ultragenyx Pharmaceutical Inc.🔗 ChEMBL
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Gene drug with a maximum clinical stage of Phase 1 2 (across all indications), with 2 investigational indications.
all catalysts(0)
nothing on the calendar yet
News(3)
- Ultragenyx wins FDA greenlight for first Sanfilippo therapy, priced at nearly $4MThe market approval for Fayuvi, which was rejected in 2025 due to manufacturing problems, comes shortly after Ultragenyx got the FDA nod for its first gene therapy last month.biospace · 15d ago
- FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type AThe U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known…fda_press · 16d ago
- Biotechs bring big launches and pharma rivalries into Q2 earnings periodWith the second quarter earnings period underway, BioSpace examines five biotechs with meaningful catalysts that analysts and investors are watching.biospace · 2mo ago
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