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Latest From uniQure N.V.
Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian
BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.
Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian
BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.
Gene Therapy Manufacturing Hurdle: Sponsors Unwilling To Share ‘Secret Sauce’
US FDA’s Peter Marks says companies are not exchanging information about adenoviral factors, which is probably slowing down the field. He describes NIH's plans for a bespoke gene therapy consortium for vector generation and continued efforts for regulatory harmonization.
Biotech Entrepreneur Aldag Inspired By Italy's Genespire
Jörn Aldag, who has over 20 years of leadership experience in the life sciences industry, including CEO of gene therapy pioneer uniQure, tells Scrip that the research carried at the world-class Italian research institute SR-Tiget.
Company Information
- Industry
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Pharmaceuticals
- Drug Delivery
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Biotechnology
- Antisense, Oligonucleotides
- Gene Therapy, Cell Therapy
- Large Molecule
- Other Names / Subsidiaries
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- Amsterdam Molecular Therapeutics (AMT)
- InoCard GmbH
- uniQure Germany GmbH
- uniQure U.S.A.
- uniQure The Netherlands
- uniQure Inc.