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.

Click here to read the full press release.