{"schema_version":"secwatch.filing_event.v1","accession":"0000950170-24-112402","form_type":"8-K","ticker":"RARE","cik":"0001515673","company_name":"Ultragenyx Pharmaceutical Inc.","filed_at":"2024-10-03T23:59:59+00:00","discovered_at":"2026-05-14T18:03:08.652048+00:00","generated_at":"2026-05-30T21:26:43.937026+00:00","sec_items":["8.01"],"event_type":"other_material","sentiment":"positive","materiality_score":0.75,"calibrated_materiality_score":0.75,"confidence":"high","headline":"UX701 gene therapy shows clinical activity; 6 of 15 Wilson disease patients tapered off standard-of-care","bullets":["15 patients enrolled across 3 dose cohorts; 6 completely tapered off chelators/zinc therapy; 1 additional began tapering.","Non-ceruloplasmin bound copper stabilized to normal levels; ceruloplasmin-copper activity increased, indicating improved ATP7b function.","UX701 well tolerated with no unexpected treatment-emergent adverse events or significant immunologic safety events.","Company plans to enroll a fourth cohort at moderately increased dose with optimized immunomodulation regimen to enhance efficacy.","Dose selection for randomized placebo-controlled Stage 2 and long-term follow-up in Stage 3 are planned."],"urls":{"canonical":"https://secwatch.observer/filing/0000950170-24-112402","json":"https://secwatch.observer/filing/0000950170-24-112402.json","markdown":"https://secwatch.observer/filing/0000950170-24-112402.md","text":"https://secwatch.observer/filing/0000950170-24-112402.txt","edgar_index":"https://www.sec.gov/Archives/edgar/data/1515673/000095017024112402/0000950170-24-112402-index.htm","edgar_primary_document":"https://www.sec.gov/Archives/edgar/data/1515673/000095017024112402/rare-20241003.htm"},"model":{"generated_by":"deepseek-v4-flash:cloud@v2","generated_at":"2026-05-30T21:26:43.937026+00:00"},"review":{"review_status":"machine_generated","human_reviewed":false,"corrected":false,"correction_note":null,"correction_timestamp":null,"superseded_by":null,"related_filings":[]},"source_grounded_claims":[],"license":"Source filings: public domain (SEC EDGAR). Summaries (headline + bullets): CC-BY-4.0; attribute https://secwatch.observer"}