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

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Tuesday, October 02, 2018

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CAMBRIDGE, Mass. - 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,  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. Designed to be a one-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 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.

About AVR-RD-02
AVR-RD-02 is an ex 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 a state-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 create AVR-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.

About AVROBIO, Inc.
AVROBIO, Inc., is a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose. AVROBIO's lentiviral-based gene therapies employ hematopoietic stem cells that are collected from the patient and then modified with a lentiviral vector to insert a functional copy of the gene that is defective in the target disease. AVROBIO is focused on the development of its gene therapy, AVR‑RD‑01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com.

Contacts:
Investor Contact:
Christopher F. Brinzey
Westwicke Partners
339-970-2843

Media Contact:
Kathryn Morris
The Yates Network
914-204-6412

Source: AVROBIO Inc.
4.33333333333
4.3 from 3 votes
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