Ultragenyx Pharmaceutical (RARE)
BiotechSign Signal Grade
Composite of clinical trial activity, dilution exposure, publications, short interest, and insider transactions — all from verified public filings.
Pipeline Overview
- Therapeutic Focus
- Rare Diseasesrc
Clinical Trial Activity
(ClinicalTrials.gov aggregate)
ClinicalTrials.gov aggregate as of 2026-10-02. ClinicalTrials.gov
Dilution Risk
MEDIUM DILUTION RISKDilution score: 35/100 (MEDIUM), 11.7 months cash
Derived from verified regulatory filings. Measures dilution exposure from warrants, convertibles, and offering history. DilutionWatch SEC-derived score
Short Interest
Short interest: 17.54% of float, squeeze risk: MODERATE
FINRA short sale data. FINRA short interest
Recent News & SEC Filings
- Ultragenyx shares rise after agreeing to sell FDA priority review voucher for $210 million
- Ultragenyx Lands $210M Deal To Sell FDA Priority Review Voucher – Retail Sees ‘Huge’ Buying Opportunity
- Ultragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $210 Million
- Ultragenyx (RARE) EMA Validation of MPS IIIA Gene Therapy: Inflection Point or Incremental Milestone?
- Ultragenyx's Gene Therapy for Sanfilippo Syndrome Wins EMA Validation
- Ultragenyx Announces Marketing Authorisation Application (MAA) Submission to the European Medicines Agency (EMA) for the First Investigational Gene Therapy for MPS IIIA (Sanfilippo Syndrome Type A)
- RARE Stock Climbs This Week As Investors Look Past Trial Failure To Gene Therapy Launches, Trial Results
- FDA Approves Fayuvi. What Does It Mean for Ultragenyx (RARE)’s 2027 Profitability Target?
Headlines from public sources. BiotechSign does not endorse or verify linked content.
Recent Publications
- Aortic valve stenosis in 2026: from emerging mechanisms to clinical drug development. — Atherosclerosis 2026 Sep PMID 42580147
- Lp(a) testing and treatment with Lp(a)-lowering therapies in the primary and secondary prevention of cardiovascular disease in high income countries: A preliminary health technology assessment. — Atherosclerosis 2026 Sep PMID 42526419
- Global Burden of Elevated LDL-C: Findings From the Global Burden of Disease Study 2023. — JAMA 2026 Aug 25 PMID 42525403
- Digitally Enabled Quality Improvement Intervention and LDL-C Control in Atherosclerotic Cardiovascular Disease: The SAPPHIRE-LDL Cluster Randomized Clinical Trial. — JAMA Cardiol 2026 Sep 1 PMID 42485013
- Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses. — J Neurol 2026 Jul 17 PMID 42467089
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