Ultragenyx Pharmaceutical Inc.
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Latest From Ultragenyx Pharmaceutical Inc.
Kyowa Kirin’s North America Expansion Progresses With Crysvita Commercial Takeover
The Japanese pharma has taken over commercialization of the rare disease drug from Ultragenyx under a planned transition and as part of a broader growth strategy in North America.
Product Quality ‘Wild Card’? Sarepta’s Gene Therapy Manufacturing Process Change Means More Empty Capsids
SRP-9001 intended for commercialization is not analytically comparable to the product used in earlier clinical studies; FDA CMC reviewer said agency does not mandate the extent of empty or full capsids but, instead, looks at the totality of data to assess safety and efficacy.
Deal Watch: Dianthus Will Reach Public Markets Via Reverse Merger With Stalled-Out Magenta
After shutting down its trial for a leukemia conditioning agent due to a patient death, Magenta’s review of strategic alternatives concludes with a combination with complement inhibitor-focused Dianthus.
Supplemental Filings: Pink Sheet Notebook On Disease Lobbying, Dan Leonard’s Next Act, And More
Quick takes on Ultragenyx’s CEO lobbing the US FDA for rare disease policy changes, the agency naming a new deputy assistant commissioner for media affairs, the former CEO of AAM starting a consulting firm, and more.
- Gene Therapy, Cell Therapy
- Other Names / Subsidiaries
- Dimension Therapeutics
- GeneTx Biotherapeutics LLC
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