AVROBIO Receives No Objection to Clinical Trial Application from Health Canada for AVR-RD-02 Gene Therapy for Gaucher Disease

AVROBIO (Nasdaq:AVRO) a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose, today announced that it has received no objection to its clinical trial application (CTA) from Health Canada for a Phase 1/2 clinical trial of AVR-RD-02, the Company’s gene therapy for Gaucher disease. AVR‑RD-02 is an ex vivo lentiviral gene therapy.

As quoted in the press release:

“We are excited to have achieved this regulatory milestone as a step forward in our activities to move AVR-RD-02, the next gene therapy candidate in our pipeline, into the clinic in 2019,” said Geoff MacKay, President and CEO of AVROBIO. “We believe there is a significant opportunity for gene therapy to offer a potential cure for a range of lysosomal storage diseases, and we are building a pipeline of product candidates to bring this new treatment paradigm to patients.”

The Phase 1/2 clinical trial of AVR-RD-02 (the GAU-201 Study¹) is planned to enroll 8 to 16 patients with Type 1 Gaucher disease, and will use an adaptive trial design that includes both patients that are currently receiving enzyme replacement therapy (ERT) and ERT-naïve patients. Patients will not receive ERT during the clinical trial. All enrolled patients will receive a single treatment with AVR-RD-02 and will be followed for 52 weeks to measure safety and efficacy. Efficacy endpoints for the GAU-201 Study will include enzyme activity, liver and spleen volumes, hemoglobin, platelet counts, bone mineral density, and other parameters associated with Gaucher disease.

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