Ultragenyx Pharmaceutical Inc.
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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.
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.
Investors keep piling into biotech stocks and British companies have been reaping the benefits this week.
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.
- Gene Therapy, Cell Therapy
- Other Names / Subsidiaries
- Dimension Therapeutics