Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as scAAV9.U1a.hSGSH, ABO-102, rebisufligene etisparvovec
UX111 · 1 trial · 4 indications
Exposure is defined as the time-normalized area under the curve (AUC) of the percentage reduction from baseline.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 Low Dose | EXPERIMENTAL | Dose of 0.5 X 10\^13 vg/kg |
| Cohort 2 Mid Dose | EXPERIMENTAL | Dose of 1 X 10\^13 vg/kg |
| Cohort 3 High Dose | EXPERIMENTAL | Dose of 3 X 10\^13 vg/kg |
| Cohort 4 High Dose (Spain Only) | EXPERIMENTAL | Dose of 3 X 10\^13 vg/kg |
| Name | Type | Description |
|---|---|---|
| UX111 | BIOLOGICAL | Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) will be delivered one time through a venous catheter inserted into a peripheral limb vein. |
| Prophylactic Immunomodulatory (IM) Therapy | DRUG | - |
| Optimized Prophylactic IM Therapy | DRUG | - |
| Adjuvant IM Therapy | DRUG | The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive adjuvant IM therapy. |
Inclusion Criteria: * Diagnosis of MPS IIIA confirmed by the following methods: * No detectable or significantly reduced SGSH enzyme activity by leukocyte assay, and * Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene (based upon review of docume...
UX111 is an investigational gene therapy being developed for Mucopolysaccharidosis IIIA, also known as MPS IIIA or Sanfilippo syndrome type A. It is designed to address the underlying genetic cause of this rare, progressive lysosomal storage disorder. It is not yet approved for commercial use.
UX111 targets the SGSH gene, which encodes the enzyme N-sulfoglucosamine sulfohydrolase. In MPS IIIA, mutations in SGSH lead to enzyme deficiency and accumulation of heparan sulfate. UX111 is a monoclonal antibody-based gene therapy intended to deliver a functional copy of the SGSH gene.
UX111 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on rare and ultra-rare diseases. Ultragenyx trades on the Nasdaq under the ticker symbol RARE.
UX111 is in Phase 2 clinical development. It has not been approved by the FDA and remains investigational. The ongoing trial is a Phase I/II/III study, but the overall development stage is Phase 2.
UX111 is being evaluated in a single active clinical trial, NCT02716246, titled Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH. The trial is currently recruiting participants with MPS IIIA and has an estimated enrollment of 36 patients across the United States, Australia, and Spain.
UX111 has received four FDA designations: Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease. These designations are intended to support and expedite the development of therapies for serious rare conditions.