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rAAV1-CB-hAAT · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Low dose | EXPERIMENTAL | rAAV1-CB-hAAT at dosage level of 6 x 10e11 vg/kg |
| Middle dose | EXPERIMENTAL | rAAV1-CB-hAAT at dosage level of 1.9 x 10e12 vg/kg |
| High dose | EXPERIMENTAL | rAAV1-CB-hAAT at dosage level of 6 x 10e12 vg/kg |
| Name | Type | Description |
|---|---|---|
| rAAV1-CB-hAAT | DRUG | Recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin |
Inclusion Criteria: 1. Have a diagnosis of AAT-deficiency, as defined by a serum AAT level of less than 11 µM and a phenotype or genotype either homozygous for PI\*Z or compound heterozygous consisting of PI\*Z and another allele known to be associated with disease 2. Be at least 18 and not more th...
rAAV1-CB-hAAT is an investigational gene therapy being developed for the treatment of Alpha-1 Antitrypsin Deficiency, a rare genetic condition. It is currently in Phase 2 clinical development and has not been approved by the FDA. The drug is designed to deliver a functional copy of the alpha-1 antitrypsin gene to patients.
rAAV1-CB-hAAT is being developed by Elevance Health, Inc., a company traded on the stock exchange under the ticker symbol ELV. The company is conducting clinical research on this investigational therapy for Alpha-1 Antitrypsin Deficiency, a rare disease indication.
rAAV1-CB-hAAT is currently in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. The therapy is being studied for the treatment of Alpha-1 Antitrypsin Deficiency, and its Phase 2 trial has been completed.
rAAV1-CB-hAAT has been studied in one completed Phase 2 clinical trial with the identifier NCT01054339. The trial, titled 'Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency,' enrolled 9 participants in the United States and Ireland. The study was controlled but not randomized or double-blinded.
rAAV1-CB-hAAT is a gene therapy designed to deliver a functional copy of the alpha-1 antitrypsin (AAT) gene to patients with Alpha-1 Antitrypsin Deficiency. By introducing this gene, the therapy aims to restore production of the AAT protein, which is deficient in affected individuals. The drug uses an adeno-associated virus (AAV) vector for delivery.