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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 alpha-1 antitrypsin deficiency, a rare inherited disorder. It is designed to deliver a corrected copy of the gene responsible for producing alpha-1 antitrypsin. It is in Phase 2 clinical development and is not approved for any indication.
rAAV1-CB-hAAT is being studied as a treatment for alpha-1 antitrypsin deficiency, a genetic condition in which the body produces insufficient or defective alpha-1 antitrypsin protein. The goal is to provide a functional copy of the gene so patients can produce the protein themselves. It remains investigational.
rAAV1-CB-hAAT is being developed by Elevance Health, Inc., which trades on the New York Stock Exchange under the ticker symbol ELV. The company is the sponsor of the clinical program for this gene therapy candidate.
rAAV1-CB-hAAT is in Phase 2 clinical development. One Phase 2 trial has been completed, and there are currently no active trials listed for this candidate. It is an investigational gene therapy and has not been approved by the FDA for any indication.
rAAV1-CB-hAAT has been evaluated in a single Phase 2 trial, NCT01054339, titled Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency. The trial enrolled 9 participants, was controlled, and has been completed. It was conducted in the United States and Ireland.