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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 being studied for Sanfilippo Syndrome B, a rare inherited lysosomal storage disorder also known as mucopolysaccharidosis type IIIB. It has been evaluated in a Phase 1 clinical trial in children with the condition. It is not an approved treatment.
rAAV2/5-hNAGLU is an investigational gene therapy developed by uniQure N.V. (ticker QURE). It uses an adeno-associated viral vector to deliver a corrected copy of the NAGLU gene, which encodes the enzyme alpha-N-acetylglucosaminidase. It has been studied in Sanfilippo Syndrome B and in Acute Intermittent Porphyria under a related vector construct.
rAAV2/5-hNAGLU is being developed by uniQure N.V., a gene therapy company listed under the ticker QURE. uniQure sponsored the Phase 1 clinical trial evaluating the vector in children with Sanfilippo Syndrome B. The company also sponsored a separate Phase 1 trial of the related vector rAAV2/5-PBGD in Acute Intermittent Porphyria.
rAAV2/5-hNAGLU is in Phase 1 clinical development. The Phase 1 trial in Sanfilippo Syndrome B has been completed, and no active trials of this vector are currently listed. It is an investigational gene therapy and has not been approved by the FDA or any other regulatory agency.
rAAV2/5-hNAGLU has been evaluated in one completed Phase 1 trial, NCT03300453, titled Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome. That trial enrolled 4 participants in France. A separate completed Phase 1 trial, NCT02082860, studied the related vector rAAV2/5-PBGD in Acute Intermittent Porphyria in Spain.