Scrip is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction

Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

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.

Gene Therapy Rare Diseases

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.

Deals Financing

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.’

Gene Therapy Review Pathway

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. 

Restructuring Financing
See All

Company Information

  • Industry
  • Biotechnology
  • Pharmaceuticals
  • Biotechnology
    • Gene Therapy, Cell Therapy
    • Large Molecule
      • Antibodies
  • Other Names / Subsidiaries
    • Dimension Therapeutics
    • GeneTx Biotherapeutics LLC
    • Ultragenyx
UsernamePublicRestriction

Register