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First patient with Fabry disease who received a single dose of lentiviral gene therapy achieved normal plasma α-galactosidase A enzyme activity within 45 days and has maintained those levels for six months

Data presented at 59^th Annual Meeting of Japanese Society for Inherited Metabolic Diseases

Cambridge, MA, October 12, 2017  – AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced initial six-month clinical data from the first patient treated with AVR-RD-01, its lentiviral gene therapy for Fabry disease. Fabry disease is caused by absent or deficient alpha-galactosidase A (a-Gal A) activity, a lysosomal enzyme encoded by the GLA gene.

At the initiation of the study, the patient with Fabry disease had plasma a-Gal A activity near zero. Within 45 days of receiving AVR-RD-01, the patient’s plasma a-Gal A activity increased into the normal range for individuals without Fabry. The six-month assessment demonstrated continued plasma a-Gal A activity within the normal range for individuals without Fabry. These data are from an academic Phase 1 trial being conducted in partnership with AVROBIO by the FACTs team in Canada (https://clinicaltrials.gov/ct2/show/NCT02800070 ). The data were presented at the 59^th Annual Meeting of Japanese Society for Inherited Metabolic Diseases, October 12-14, 2017, in Kawagoe, Japan.

“We are excited to share these promising data from the world’s first patient with Fabry disease to receive our gene therapy,” said Nerissa C. Kreher, MD, MBA, AVROBIO’s Chief Medical Officer. “The data demonstrate a rapid increase of a-Gal A activity from essentially zero into the normal range which has been maintained for six months. We look forward to reporting additional data from this patient and additional subjects as the clinical trial with AVR-RD-01 continues to progress.”

Summary of the data presented at the meeting include:

• Patient #1, a male subject with Fabry disease who received a single dose of AVR-RD-01, an ex vivo lentiviral gene therapy manufactured utilizing the patient’s stem cells which are genetically modified to express normally active a-Gal A.
• Within 45 days of receiving a single dose of AVR-RD-01, the Fabry patient’s plasma a-Gal A activity increased into the normal range for individuals without Fabry. At the 6-month evaluation, the plasma enzyme activity remains increased within the normal range for individuals without Fabry.
• The patient received a minimal intensity conditioning regimen prior to receiving AVR-RD-01 gene therapy. This conditioning regimen allowed for the AVR-RD-01 infusion to be performed on an out-patient basis.
• No study related serious adverse events were reported during this initial 6-month study period.

About Fabry Disease

Fabry disease is a rare genetic disorder associated with significant morbidity and early mortality, caused by defects in the GLA gene, resulting in a deficiency of the lysosomal enzyme α-galactosidase A. This enzyme is required for the breakdown of globotriaosylceramide (also known as Gb3 or GL-3). In people living with Fabry disease, Gb3 accumulates in various cells throughout the body causing the progressive clinical signs and symptoms of the disease that can lead to irreversible organ damage resulting in potentially life-threatening kidney damage, cardiac disease and early stroke. Current standard of care is enzyme replacement therapy; requiring lifelong bi-weekly infusions.

About AVR-RD-01

AVR-RD-01 is a lentiviral gene therapy being investigated as a single-dose therapy with durable and life-long potential for patients with Fabry disease. AVR-RD-01 uses a state-of-the-art lentiviral vector system that is an efficient and proven gene transfer system for the stable addition of genes to the patient’s stem cells. In patients with Fabry disease, CD34+ stem cells are extracted and isolated, and then transduced with lentiviral vector carrying a normal GLA gene to create AVR-RD-01 gene therapy. AVR-RD-01 is then infused back into the patient in an out-patient setting with the goal of restoring normal GLA gene expression such that normally active α-galactosidase A enzyme is produced by the patient’s own body.

About AVROBIO, Inc.

AVROBIO, Inc., a leader in lentiviral-based gene therapies, is a clinical-stage company developing disruptive therapies that have the potential to transform patients’ lives with a single dose. The Company is focused on the development of its clinical program in Fabry disease and pre-IND programs in Gaucher disease and Cystinosis while actively building a pipeline of therapies to treat Pompe disease and other rare and non-rare genetic diseases. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com.

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Kathryn Morris
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