AVROBIO, Inc. Announces Acceptance of Investigational New Drug (IND) Application for Investigator-Sponsored Phase 1/2 Clinical Trial for AVR-RD-04 Gene Therapy for Cystinosis

Phase 1/2 study to be led by team at University of California, San Diego, with rights licensed to AVROBIO

CAMBRIDGE, MA, USA I January 18, 2019 I AVROBIO, Inc. (NASDAQ: AVRO) (the “Company”) a Phase 2 clinical-stage gene therapy company, today announced that its investigational gene therapy candidate for cystinosis, AVR-RD-04, is cleared to begin a Phase 1/2 clinical trial, following acceptance of the investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA). The Phase 1/2 clinical trial is a planned investigator-sponsored trial to be conducted by the University of California, San Diego (UCSD) under the direction of Stephanie Cherqui, PhD, Associate Professor in the Department of Pediatrics at UCSD and a leading expert in stem cells and gene therapy for cystinosis.

AVR‑RD-04 is designed as one-time gene therapy and is being investigated for the treatment of cystinosis by inserting the functional gene for human cystinosin (CTNS), and is designed to maximize the likelihood of sustained CTNS production in patients to correct for the single-gene defect in the CTNS gene that causes cystinosis.

“We are pleased to collaborate with Dr. Stephanie Cherqui of the University of California, San Diego, and we share the vision of her and her team, and the dedicated patient advocates in the cystinosis community, to develop what we expect will be a single-dose and potentially transformative gene therapy for the treatment of people with cystinosis,” said Geoff MacKay, President and CEO of AVROBIO. “We believe there is a significant opportunity for gene therapy for a range of lysosomal storage diseases, and we are building a pipeline of product candidates to bring this new treatment paradigm to patients.”

About Cystinosis

Cystinosis is a rare, genetic, autosomal recessive, lysosomal storage disease caused by the accumulation of the amino acid cystine. Cystine crystals build up and cause complications in many organs and tissues. The most severe form of cystinosis begins in infancy, causing poor growth and a particular type of kidney damage. Untreated children with cystinosis may experience kidney failure, and other symptoms include muscle deterioration, blindness, inability to swallow, diabetes, thyroid and nervous system problems. More than 90% of untreated patients require a kidney transplant before the age of 20. It is estimated that cystinosis disease is diagnosed in approximately one in 170,000 people.

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


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