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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 the treatment of MPS IIIA, also known as Sanfilippo syndrome type A. It is designed to address the underlying genetic cause of this rare disease. The drug is currently in Phase 2 clinical development and has not yet been approved by regulatory authorities.
UX111 is a gene therapy that delivers a functional copy of the SGSH gene using an adeno-associated viral vector. The SGSH gene provides instructions for producing the enzyme N-sulfoglucosamine sulfohydrolase, which is deficient in patients with MPS IIIA. This enzyme is essential for breaking down certain complex carbohydrates in the body.
UX111 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol RARE. Ultragenyx focuses on developing therapies for rare and ultrarare diseases. The company is conducting clinical trials to evaluate the safety and efficacy of UX111 in patients with MPS IIIA.
UX111 is currently in Phase 2 clinical development. It is being evaluated in an ongoing Phase I/II/III gene transfer clinical trial. The drug has received several FDA designations, including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, Rare Pediatric Disease, and Accelerated Approval, reflecting its potential to address a serious unmet medical need.
UX111 is being studied in a single clinical trial registered as NCT02716246, titled 'Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH'. This trial is currently recruiting participants with MPS IIIA, Sanfilippo syndrome, and related conditions. The study is enrolling up to 36 patients across the United States, Australia, and Spain.
Yes, UX111 is also known as scAAV9.U1a.hSGSH, which is the name used in the clinical trial title. This designation refers to the specific gene therapy construct being tested. The trial NCT02716246 is evaluating this therapy in patients with MPS IIIA and Sanfilippo syndrome.