AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Cystinosis

Cystinosis Program Becomes Company’s Fourth Gene Therapy
for Lysosomal Storage Disorders

Cambridge, MA, October 4, 2017 – AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to cystinosis. This program becomes AVROBIO’s fourth gene therapy for lysosomal storage disorders (LSDs), and with an anticipated IND filing later this year is expected to enter the clinic in early 2018. The cystinosis program was licensed from GenStem Therapeutics, Inc. Terms of the license agreement were not disclosed.

“This cystinosis program is a strong strategic fit with our pipeline,” said Geoff MacKay, AVROBIO’s President and Chief Executive Officer. “We are pleased to partner with Dr. Stephanie Cherqui and her team at the University of San Diego (UCSD), as well as with GenStem Therapeutics, Inc. as we share their vision of developing a novel and potentially transformative gene therapy for the treatment of patients with cystinosis.”

“We are extremely pleased to partner with AVROBIO. As a leader in gene therapies for lysosomal storage disorders, they will substantially accelerate the development and commercialize our novel treatment for cystinosis, said Jeffrey Ostrove, Ph.D., Chief Executive Officer of GenStem Therapeutics, Inc. “This will help bring a novel therapy to patients who suffer from this disease.”

Cystinosis, like all lysosomal storage disorders, is a progressive and potentially fatal metabolic disorder with an estimated incidence of 1 in 100,000 to 200,000 live births. Cystinosis is caused by a single-gene defect in the CTNS gene leading to the accumulation of an amino acid called cystine in all tissues and organs of the body including the kidneys, eyes, muscles, thyroid and pancreas. Patients with cystinosis have a wide range of symptoms depending on the age of onset and disease severity including renal Fanconi syndrome and renal failure by adolescence, sensitivity to light, blindness, severe muscle weakness, difficulty swallowing, and endocrinopathies including hypothyroidism and diabetes, and neurological defects.

The aim of AVROBIO’s investigational gene therapy to treat cystinosis is to deliver lasting and meaningful benefits for patients. The patient’s peripheral blood stem cells are extracted and genetically modified by adding a new, fully functional copy of the faulty gene. The modified cells, once delivered back into the patient via a one-time infusion, enable durable gene rescue of the progressive tissue injury, with the potential to significantly improve patient outcomes and eliminate burdensome and costly lifelong cysteamine treatments.

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 Phase 1 program in Fabry disease and pre-IND programs in cystinosis and Gaucher disease, while actively building a pipeline of therapies to treat Pompe disease and other rare and non-rare genetic diseases. AVROBIO also has an immuno-oncology gene therapy program. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com.

Media Inquiries:
Kirsten Dupuis

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