Exhibit 99.2
AVROBIO Receives No Objection to Clinical Trial Application from Health Canada
forAVR-RD-02 Gene Therapy for Gaucher Disease
Company plans to initiate Phase 1/2 clinical trial in patients with Gaucher disease in 2019
AVROBIO continues to advance its pipeline of gene therapies for lysosomal storage disorders
CAMBRIDGE, Mass.– October 1, 2018 – AVROBIO, Inc. (Nasdaq: AVRO) (the “Company”), 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 ofAVR-RD-02, the Company’s gene therapy for Gaucher disease.AVR-RD-02 is anex vivo lentiviral gene therapy. Designed to be aone-time therapy, it works by permanently integrating the GBA gene that encodes functional glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease, to enable continuous GBA production and distribution to tissues and organs.
“We are excited to have achieved this regulatory milestone as a step forward in our activities to moveAVR-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 ofAVR-RD-02 (theGAU-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) andERT-naïve patients. Patients will not receive ERT during the clinical trial. All enrolled patients will receive a single treatment withAVR-RD-02 and will be followed for 52 weeks to measure safety and efficacy. Efficacy endpoints for theGAU-201 Study will include enzyme activity, liver and spleen volumes, hemoglobin, platelet counts, bone mineral density, and other parameters associated with Gaucher disease.
AboutAVR-RD-02
AVR-RD-02 is anex vivo lentiviral gene therapy being investigated as a single-dose therapy with the potential to provide durable and life-long potential therapeutic benefit for patients with Gaucher disease.AVR-RD-02 is designed to employ astate-of-the-art lentiviral vector platform that is an efficient and proven gene transfer system for the permanent integration of a functional copy of the gene into the patient’s own stem cells. In patients with Gaucher disease, hematopoietic stem cells are collected from the patient, and then transduced with lentiviral vector carrying a functional version of the GBA gene that encodes functional GCase, the enzyme that is deficient in Gaucher disease, to createAVR-RD-02 gene therapy.AVR-RD-02 is then infused back into the patient with the goal of restoring normal GBA gene expression such that functional GCase enzyme is sufficiently produced by the patient’s own body.
| 1 | The official name of the‘GAU-201 Study’ isAVRO-RD-02-201, which is a Phase 1/2 trial of AVROBIO’s investigational gene therapy,AVR-RD-02, in Gaucher disease. |
