Avrobio announced clinical data and patient updates from the investigator-sponsored Phase 1 study and the Avrobio-sponsored Phase 2 clinical trial of AVR-RD-01. AVR-RD-01 is an ex vivo lentiviral gene therapy being investigated in Fabry disease. Designed to be a one-time therapy, it works by inserting the GLA gene that encodes functional a-galactosidase A to enable continuous AGA production and distribution to tissues and organs. The investigator-sponsored Phase 1 study is designed to assess the safety of AVR-RD-01 in up to six patients with Fabry disease who have been treated with standard of care enzyme replacement therapy, or ERT, for at least six months prior to receiving AVR-RD-01. The Phase 1 study is conducted by the FACTs team in Canada. As a Phase 1 study, the primary endpoint of this study is safety. Continued patient enrollment is planned and the protocol has been amended to allow the FACTs team, at their discretion, to discontinue ERT six months after treatment with AVR-RD-01. Preliminary safety data from these three subjects indicate AVR-RD-01 was generally well tolerated and no serious adverse events related to AVR-RD-01 have been reported. Preliminary safety data from patient FAB-201-1 indicate that AVR-RD-01 was generally well tolerated. Enrollment in the FAB-201 Study is ongoing.
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