Scrip is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

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.

Gene Therapy: AAV Doses Should Not Be Subject To Fixed Upper Limit, US FDA Panel Says

Lack of reference standards inhibits ability to make comparisons between products or set caps for total vector genome dose or total capsid dose, advisory committee says; panelists suggest other approaches to prevent and mitigate toxicities, including running longer-term animal studies and investigating the efficacy of immunosuppression prophylaxis and treatment strategies.

Advisory Committees Gene Therapy

AZ's Rare Disease Unit Reports Positive Phase III Results For Copper-Binding ALXN1840

Alexion/AstraZeneca has announced positive top-line results from the Phase III study of ALXN1840 in patients with the rare genetic disorder, Wilson disease, including in patients already on standard-of-care therapies. The compound is ahead of two candidate gene therapies from other companies at early-stage clinical development.

Rare Diseases Clinical Trials

UK Firms Big And Small Benefit From Biotech Bubble

Investors keep piling into biotech stocks and British companies have been reaping the benefits this week.

United Kingdom Financing

Scrip Asks…What Does 2021 Hold For Biopharma? Part 2: COVID-19 Shock Waves And Silver Linings

Executives expect the changes wrought by necessity in the face of a global pandemic to have lasting effects, both positive and negative, on the biopharma sector. Here they share their predictions for the coming year.

Coronavirus COVID-19 Scrip Asks
See All

Company Information

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

Register