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rAAV2/5-PBGD vector dosage 1 · 1 trial · 1 indication
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
| 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 |
| 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. |
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-PBGD vector dosage 1 is an investigational gene therapy being studied for two rare diseases: Acute Intermittent Porphyria and Sanfilippo Syndrome B. It is in Phase 1 clinical development for both conditions.
rAAV2/5-PBGD vector dosage 1 is being developed by uniQure N.V., a biopharmaceutical company traded on NASDAQ under the ticker QURE.
rAAV2/5-PBGD vector dosage 1 is in Phase 1 clinical development. It is an investigational gene therapy and has not been approved by regulatory authorities.
rAAV2/5-PBGD vector dosage 1 has been studied in two completed Phase 1 trials. NCT02082860 enrolled 8 patients with Acute Intermittent Porphyria in Spain, and NCT03300453 enrolled 4 children with Sanfilippo Syndrome B in France.
rAAV2/5-PBGD vector dosage 1 is a gene therapy, using an adeno-associated viral vector to deliver the PBGD gene. It is being developed for rare diseases.