Ultragenyx Pharmaceutical Inc.
http://www.ultragenyx.com/
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Latest From Ultragenyx Pharmaceutical Inc.
BioMarin Prepares Investors For A Possible US Hemophilia Gene Therapy Delay
FDA review of the company's BLA for a hemophilia gene therapy could be delayed by three months to allow the agency to review a three-year data analysis from an ongoing trial.
Financing Quarterly Statistics, Q3 2022
During Q3, biopharmas brought in an aggregate $18.1bn in financing and device company fundraising totaled $5.1bn; while in vitro diagnostic firms and research tools players raised $771m.
Accelerated Approval For Gene Therapies: FDA’s Wilson Bryan Offers A ‘Framework’
Bryan, who oversees reviews of gene and cell therapies in US FDA’s biologics center, cautioned against falling in love with a biomarker when it is showing no ‘clinical effect.’
Finance Watch: With Limited Fundraising Options, Biotechs Restructure To Extend Cash Runways
Mereo will reduce its headcount by 40% to focus on its lead programs while NeuBase revealed a 60% workforce reduction to extend its cash runway. Also, Invivyd (formerly Adagio), Greenlight and BioMarin announced job cuts, while Otonomy and others planned strategic shifts.
Company Information
- Industry
- Biotechnology
- Pharmaceuticals
-
Biotechnology
- Gene Therapy, Cell Therapy
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Large Molecule
- Antibodies
- Other Names / Subsidiaries
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- Dimension Therapeutics
- GeneTx Biotherapeutics LLC
- Ultragenyx
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