Wednesday, July 8, 2026

pharma · Cystic Fibrosis · Multiple Sclerosis · RARE

Ultragenyx UK

UGenDx is a pharma organization headquartered in NOVATO, CA, GB. It trades on NYSE under ticker RARE. Primary therapeutic focus areas include Cystic Fibrosis, Multiple Sclerosis, Fanconi's Anemia, Hereditary Inclusion Bo

Newcastle upon tyne, NE128RL, GB, NOVATO, CA HQ
2024 Founded
2 Employees
Public company Type
RARE · NYSE Ticker
Company details
Status
Public
HQ
Newcastle upon tyne, NE128RL, GB, NOVATO, CA
Founded
2024
Employees
2
Programs
201
Drugs
168
Patents
23
Clinical program

DTX401

Phase 3 · gene therapy · Glycogen Storage Disease Type IA

DTX401 for Glycogen Storage Disease Type IA is a Phase 3 program (gene_therapy) at Ultragenyx UK Limited with 1 ClinicalTrials.gov record(s).

← All Ultragenyx UK Limited projects Phase 3 gene therapy completed

Internal code DTX401-CL301

Full intelligence profile for this program is being prepared.

At a glance

Sponsor
Ultragenyx UK Limited
Phase
Phase 3
Modality
gene_therapy
Indication
Glycogen Storage Disease Type IA
Status
completed
Trials
1