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Also known as AAV5
rAAV2/5-hNAGLU · 2 trials · 2 indications
To assess the safety and determine the maximum therapeutic safe dose of the investigational gene therapy (GT) product rAAV2/5-PBGD for the treatment of AIP, registering and evaluating the occurrence of Adverse Events and/or Serious Adverse Events at the dose identified will be performed
Multiple measurements will be aggregated to derive the number of participants with Abnormal Laboratory Values and/or Adverse Events that are related to Treatment.
| Arm | Type | Description |
|---|---|---|
| Cohort A | EXPERIMENTAL | rAAV2/5-PBGD vector dosage 1 |
| Cohort B | EXPERIMENTAL | rAAV2/5-PBGD vector dosage 2 |
| Cohort C | EXPERIMENTAL | rAAV2/5-PBGD vector dosage 3 |
| Cohort D | EXPERIMENTAL | rAAV2/5-PBGD vector dosage 4 |
| rAAV2/5-hNAGLU | EXPERIMENTAL | Each patient will receive 960 µL of vector suspension. The vector suspension will be deposited simultaneously at 16 sites, each deposit containing 2.4x 1011 vg (4x1012 vg in total). |
| Name | Type | Description |
|---|---|---|
| rAAV2/5-PBGD vector dosage 1 | GENETIC | Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. |
| rAAV2/5-PBGD vector dosage 2 | GENETIC | Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. |
| rAAV2/5-PBGD vector dosage 3 | GENETIC | Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. |
| rAAV2/5-PBGD vector dosage 4 | GENETIC | Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1. |
| rAAV2/5-hNAGLU | DRUG | one-time brain intraparenchymal gene therapy dose |
Inclusion Criteria: * Patient's written Informed Consent * Age between 18 and 64 years, inclusively. * Patients with confirmed diagnosis of Acute Intermittent Porphyria(AIP), as confirmed by clinical, biochemical data and genetic confirmation of porphobilinogen deaminase (PBGD) gene mutation. The p...
rAAV2/5-hNAGLU is an investigational gene therapy developed by uniQure N.V. for Sanfilippo Syndrome B, a rare inherited metabolic disorder. It is designed to deliver a functional copy of the human NAGLU gene using an AAV2/5 viral vector, with the goal of restoring the enzyme missing in affected patients.
rAAV2/5-hNAGLU is being studied for the treatment of Sanfilippo Syndrome B, also known as Mucopolysaccharidosis type IIIB, a rare lysosomal storage disorder. It is administered as an intracerebral gene therapy in children with the condition, aiming to address the underlying enzyme deficiency in the central nervous system.
rAAV2/5-hNAGLU is developed by uniQure N.V., a gene therapy company listed on Nasdaq under the ticker QURE. The company is conducting clinical development of this vector for Sanfilippo Syndrome B.
rAAV2/5-hNAGLU is in Phase 1 clinical development. The completed Phase 1 trial evaluated intracerebral gene therapy in children with Sanfilippo Syndrome B. The program remains investigational and has not been approved by the FDA or any regulatory authority.
rAAV2/5-hNAGLU has been evaluated in one completed Phase 1 trial, NCT03300453, titled Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome. The trial enrolled 4 patients in France. No active trials of this vector are currently listed.