RARE · Ultragenyx Pharmaceutical Inc. — research history
Complete research history. Every dossier, draft, kill, publish, and lesson the system has produced on RARE. Public so users can audit, AI can re-reference. Live price refreshes every 60s.
About RARE · Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701, an adeno-associated AAV9 gene therapy which is in Phase 2 clinical trial for the treatment of Wilson liver disease. The company has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Regeneron; and Abeona. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
Live Quote
HOLD (score +1) · 12-1 mom -36.2% · RSI 69.6 · above_both · -30.6% from high
Targets blend Wall Street consensus (19 analysts: low $25.00 / mean $52.05 / high $96.00) with chart-derived floors and ceilings.
1-Year Chart · RSI · MACD
Research Timeline
Newest first. Each entry shows what stage produced it, the verdict/decision, and the reasoning.
recent analyst:skip 0.0h ago < cooldown 168h, no material change
Active anti-signal gates (going concern, dilution, accounting) combined with $800M annual cash burn, negative FCF, and no near-term catalysts make this a high-risk biotech with insufficient edge for publication.
Ultragenyx is a rare-disease biotech that has cratered 78% over five years to near the bottom of its 52-week range. The trigger — three Form 4 filings in 14 days — turns out to be RSU vest+sell activity, NOT open-market purchases (code A for grants, S for tax-withholding sales; no P-codes found). There are ZERO confirmed CEO/CFO/Director open-market buys in the lookback window. However, two high-impact catalysts exist: UX111 PDUFA Sep 19, 2026 and DTX401 PDUFA Aug 23, 2026 — both gene therapies for fatal pediatric diseases with no approved alternatives. Analyst consensus sits at $51.52 vs current $24.19 (~113% upside). The company is cash-rich ($534M liquid), guiding to $730-760M revenue in 2026 and profitability in 2027, but burns ~$800M/year in operating cash flow. The stock presents a bounded asymmetric setup around the PDUFA events: binary on approval, but heavily discounted given five-year price destruction.