Ultragenyx Pharmaceutical submitted a Rolling BLA for its DTX401 gene therapy to the FDA.
2 reports, 1 independent
Updated Aug 24
Gone quiet
- Reports
- 2
- Developments
- 1
- Repetition
- 50%
New informationRepeats or wire copies
AI-generated analysis. Brind wrote this summary from the reports listed below. It can be wrong. Each section says how much you can rely on it, and the sources are linked so you can check.
What happened
Ultragenyx Pharmaceutical submitted a Rolling BLA for its DTX401 gene therapy to the FDA.
Who's involved
What this event is mainly aboutKeep exploring
The entities involved
- FDA
-
Ultragenyx Pharmaceutical
biopharmaceutical company based in Novato, California, United States
Nothing else this week.
Related events
- The FDA reviewed gene therapy applications and provided milestone payments for dosing involving Regenxbio and AbbVie.
- uniQure N.V. traded on the Nasdaq stock exchange and submitted a Biologics License Application (BLA) for accelerated approval of its gene therapy.
- Solid Biosciences received FDA approval for a gene therapy trial on July 9, 2025.
Coverage
Newest first; wire copies grouped- Simply Wall St.Ultragenyx Pharmaceutical (RARE) Valuation Spotlight as DTX401 Gene Therapy Advances With FDA Submission Ultragenyx Pharmaceutical (RARE) just kicked off a rolling Biologics License Application submi
- Simply Wall St.Will Ultragenyx’s (RARE) Rolling FDA Submission for DTX401 Reshape Its Rare Disease Narrative? Earlier this week, Ultragenyx Pharmaceutical announced it has started a rolling submission of a Biologic