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Ultragenyx Pharmaceutical Inc

New Haven, Connecticut

Oncologygene therapyPhase IIIListed · RARE

This company trades under its own ticker, so it sits outside Biodatica’s pre-ticker window. The record stays here because its filings are real, but the feed leaves listed companies out unless you ask for them.

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What changed

Phase III trial registered: Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

2d agoTrial registeredJul 17, 2026Source filing (opens the source filing in a new tab)

Momentum

99no change

Momentum 99 out of 100, unchanged since the last recompute.

Accelerating: several high-weight events landed recently.

Runway (estimate)

—unknown

No disclosed raise on file, so there is nothing to model from.

Stage

Phase III

The furthest-along thing in the public record. Not a projection.

Runway is an estimate: the last disclosed raise (SEC Form D) minus a modeled burn rate for the company’s stage and therapeutic area. It is not a disclosure, and companies routinely raise non-dilutive money we can’t see. How this is calculated.

Grant to raise

Every record Biodatica has resolved onto this company, newest first, each linked to the filing it came from. Where a source publishes no stable deep link for a record, the identifier is shown so you can look it up yourself.

  1. Trial

    PHASE3 registered

    Dec 3, 2024

    Angelman Syndrome · ACTIVE_NOT_RECRUITING · sponsor: Ultragenyx Pharmaceutical Inc · Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

    ClinicalTrials.gov · NCT06617429 (opens the source filing in a new tab)

  2. Trial

    PHASE3 registered

    Feb 28, 2023

    Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) · ACTIVE_NOT_RECRUITING · sponsor: Ultragenyx Pharmaceutical Inc · A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

    ClinicalTrials.gov · NCT05933200 (opens the source filing in a new tab)

  3. Trial

    Study registered

    Jan 31, 2022

    Tumor-induced Osteomalacia (TIO) · ACTIVE_NOT_RECRUITING · sponsor: Ultragenyx Pharmaceutical Inc · Tumor-induced Osteomalacia Disease Monitoring Program

    ClinicalTrials.gov · NCT04783428 (opens the source filing in a new tab)

  4. Trial

    PHASE3 registered

    Nov 8, 2021

    Glycogen Storage Disease Type IA · COMPLETED · sponsor: Ultragenyx Pharmaceutical Inc · A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

    ClinicalTrials.gov · NCT05139316 (opens the source filing in a new tab)

  5. Trial

    PHASE1/PHASE2 registered

    Feb 24, 2020

    Angelman Syndrome · COMPLETED · sponsor: Ultragenyx Pharmaceutical Inc · A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

    ClinicalTrials.gov · NCT04259281 (opens the source filing in a new tab)

  6. Trial

    Study registered

    Jul 15, 2019

    Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia) · COMPLETED · sponsor: Ultragenyx Pharmaceutical Inc · Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

    ClinicalTrials.gov · NCT03970278 (opens the source filing in a new tab)

  7. Trial

    PHASE2/PHASE3 registered

    Apr 25, 2016

    MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III · RECRUITING · sponsor: Ultragenyx Pharmaceutical Inc · Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

    ClinicalTrials.gov · NCT02716246 (opens the source filing in a new tab)

Biodatica publishes impersonal research shown identically to every subscriber. This dossier is informational only, it is not investment advice, and the momentum and runway figures are estimates derived from public filings. The underlying records are U.S. government works. Disclosures · Data sources