FINNENCE ▪ THE WIRE FILE
THE WIRE · № 79778
Company■ Mixed
Phase 3 results posted: A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) — Ultragenyx Pharmaceutical Inc
KINDCompany
DIRECTION■ Mixed
SOURCES1
ENTITIES2
STORY SIZESTANDALONE
FIRST SEEN
LAST UPDATE
STATEactive
01 THE MACHINE READ
WHY IT MATTERS.
Phase 3 results for a gene therapy could shift sentiment on Ultragenyx and rare-disease peers.
Gene therapy data point for rare disease
CATALYST TO WATCH
Company interpretation and regulatory next steps
MAIN RISK
Results direction not stated in text
FULL DISPATCH
Sponsor: Ultragenyx Pharmaceutical Inc; Status: Completed; Conditions: Glycogen Storage Disease Type IA; Interventions: DTX401, Placebo, Oral prednisolone; Enrollment: 52 (actual); First posted: 2021-12-01; Results posted: 2026-10-14; NCT: NCT05139316
02 THE STORY SO FAR
THE RECORD, IN ORDER.
STANDALONE ITEM — NOT YET FOLDED INTO A DEVELOPING STORY.
03 WHO'S INVOLVED
ENTITIES ON FILE.
AFFECTEDHealthcare
AFFECTEDSPDR Biotech ETF · XBI
04 RECEIPTS
THE SOURCE RECORD.
1 SOURCES CONFIRMED05 PROPAGATION
WHAT THIS TOUCHES.
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KEEP READING THE MACHINE
Summaries are generated and may contain errors — every claim links its sources.