RARE (Ultragenyx Pharmaceutical Inc.) stock: $14.51, $1.43B market cap, $292.0M cash, $1.14B EV, $1.43B fully diluted market cap. Next expected readout: Jan-27. Lead program: setrusumab (Osteogenesis Imperfecta), Phase 3. Data from Fully Diluted's biotech stocks database.
Pipeline assets
Crysvita
Approved · Active Modality: fully human monoclonal antibody
Target FGF23
Partnerships Kyowa Kirin (KKC): collaboration and license agreement (Aug 2013, as amended); KKC manufactures/supplies
Indications- X-linked hypophosphatemia (XLH), adults and pediatric 1+ (Approved)
- Tumor-induced osteomalacia (TIO) (Approved)
Status note Targets FGF23; subcutaneous injection; approved in US, EU and certain other regions.
Evkeeza
Approved · Active Modality: fully human monoclonal antibody (IV)
Target ANGPTL3
Indications- Homozygous familial hypercholesterolemia (HoFH) (Approved)
Status note Binds and blocks ANGPTL3; approved product.
Dojolvi
Approved · Active
Indications- Japan (NHI listed; launched May 2026 under Conditional Approval System) (Approved)
Status note Listed on Japan National Health Insurance drug price list and officially launched May 2026 after manufacturing and marketing approval Mar 23, 2026 under the Conditional Approval System for Pharmaceuticals.
DTX401
Phase 3 · Active Modality: AAV8 gene therapy (one-time IV infusion)
Aliases pariglasgene brecaparvovec
Target G6Pase-α (G6PC)
Indications- Glycogen Storage Disease Type Ia (GSDIa) (Phase 3)
Status note Designed to deliver stable expression and activity of G6Pase-α. BLA accepted for review Feb 2026 with Priority Review; FDA informed company (Apr 2026) an Advisory Committee meeting was planned.
UX111
Phase 3 · Active Modality: AAV9 gene therapy (one-time IV infusion)
Aliases ABO-102
Target SGSH (sulfamidase)
Partnerships Abeona Therapeutics (exclusive license agreement, announced May 2022)
Indications- Sanfilippo syndrome Type A (MPS IIIA) (Phase 3)
Status note Resubmitted BLA accepted Apr 2026 after prior CRL.
UX701
Phase 3 · Active Modality: AAV9 gene therapy (single IV infusion)
Target ATP7B
Partnerships REGENXBIO Inc. (license agreement)
Indications- Wilson disease (pivotal Cyprus2+ study) (Phase 3)
Status note Delivers stable expression of truncated ATP7B copper transporter to improve copper distribution/excretion and reverse Wilson liver disease pathology. Ongoing pivotal Cyprus2+ study; Stage 1 dose-finding across four sequential dose cohorts (Cohort 4 enrollment of 5 patients completed Sep 2025).
DTX301
Phase 3 · Active Modality: AAV8 gene therapy (one-time IV infusion)
Target OTC
Indications- Ornithine transcarbamylase (OTC) deficiency (Enh3ance Phase 3) (Phase 3)
Status note Enh3ance study continues; second primary endpoint readout at 64 weeks of follow-up.
GTX-102
Phase 3 · Active Modality: antisense oligonucleotide (ASO; intrathecal)
Target UBE3A
Partnerships GeneTx Biotherapeutics LLC (license agreement)
Indications- Angelman syndrome (Aspire Phase 3) (Phase 3)
- Angelman syndrome, other genotypes/ages (Aurora Phase 2/3) (Phase 2)
Status note Inhibits expression of the paternal UBE3A antisense transcript. Aspire Phase 3 (129 patients aged 4-17 with full maternal UBE3A deletion, 48 weeks) fully enrolled Jul 2025. Aurora Phase 2/3 in other genotypes/ages: enrollment begun Oct 2025, expected complete H2 2026.
UX143
Phase 3 · Active Modality: fully human monoclonal antibody
Target sclerostin
Partnerships Mereo BioPharma 3 (Mereo): collaboration agreement
Indications- Osteogenesis imperfecta (OI) (Phase 3)
Status note Dec 2025: Phase 3 Orbit and Cosmic studies did not achieve their primary endpoints. Feb 2026: restructuring curtailed UX143 manufacturing. No discontinuation of development disclosed.
UX016
Preclinical · Active Modality: small molecule prodrug of sialic acid
Indications- GNE myopathy (Preclinical)
Status note Substrate replacement therapy for GNE myopathy; FDA cleared IND application Mar 2026; program is funded.
Per-asset detail extracted from the 10-Q filed 2026-08-05. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.