Ultragenyx Pharmaceutical Inc.
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Latest From Ultragenyx Pharmaceutical Inc.
Partnered with AbbVie on a wet AMD gene therapy, Regenxbio is also advancing wholly owned candidates for Duchenne muscular dystrophy and MPS I and MPS II.
Unsuccessful attempts to use real-world evidence for approvals highlight the need to demonstrate relationship between real-world data and clinical trial patients. The Pink Sheet examines what went wrong for Bluebird/Bristol’s Abecma, Ultragenyx’s Dojolvi, and Karyopharm’s Xpovio.
Investigational products from Ultragenyx and MinervaX secured a place on the European Medicines Agency’s priority medicines scheme this month.
Ultragenyx is buying its Angelman syndrome partner GeneTx for $75m on the heels of an interim Phase I/II readout that shows signs of clinical activity and appears to resolve earlier safety worries.
- Gene Therapy, Cell Therapy
- Other Names / Subsidiaries
- Dimension Therapeutics
- GeneTx Biotherapeutics LLC