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Latest From uniQure N.V.
The company is aiming for an outcomes-based payment model in the US, while it is confident the gene therapy will fare better than some others have in Europe.
The therapy could also face competition from players like Freeline, but new Phase III data position uniQure and CSL Behring well for planned FDA and EMA filings.
There have been some recent successes on the accelerated assessment front in the EU but more requests are rejected than granted.
A bluebird executive spoke during ARM’s Cell and Gene Meeting On The Mesa about why the firm pulled Zynteglo out of the EU, but while progress has been made in the US, long-term challenges remain.
- Drug Delivery
- Antisense, Oligonucleotides
- Gene Therapy, Cell Therapy
- Large Molecule
- Other Names / Subsidiaries
- Amsterdam Molecular Therapeutics (AMT)
- InoCard GmbH
- uniQure Germany GmbH
- uniQure U.S.A.
- uniQure The Netherlands
- uniQure Inc.