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

Preclinical Proof-of-Concept Achieved, IND-Enabling Activities Underway

Cambridge, MA, March 22, 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 Gaucher disease. This late-stage preclinical program becomes AVROBIO’s second therapy for lysosomal storage disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program. The program for Gaucher disease was licensed from Dr. Stefan Karlsson at Lund University in Sweden, where preclinical proof-of-concept has been demonstrated. Terms of the license agreement were not disclosed.

“The addition of a gene therapy targeting an LSD with compelling preclinical proof-of-concept is an excellent strategic fit for AVROBIO,” said Geoff MacKay, AVROBIO’s President and Chief Executive Officer. “Our talented team of industry veterans enables us to accelerate the development of this program with the ultimate goal of benefiting Gaucher disease patients worldwide. IND-enabling activities are currently underway in preparation for a Phase 1/2 clinical study. We are excited to add this program to our portfolio as we continue to focus on building our pipeline of gene therapies to treat rare diseases.”

Gaucher disease is a genetic disorder in which a fatty substance called glucosylceramide accumulates in certain tissues and organs. The disorder is caused by a hereditary deficiency of the enzyme glucocerebrosidase, and typically impacts the spleen, liver, lungs and bones. Gaucher disease is one of the most common LSDs, occurring in up to 1 in 40,000 live births in the general population.

The aim of AVROBIO’s investigational gene therapy to treat Gaucher disease is to deliver lasting and meaningful benefits for patients. The patient’s blood stem cells are extracted and genetically modified by adding a new, fully functional copy of the faulty gene. The modified cells are then delivered back into the patient via a one-time infusion. A durable elevation of endogenous enzyme is expected, with the potential to significantly improve patient outcomes and eliminate burdensome and costly lifelong biweekly intravenous infusions of enzyme replacement therapy.

The Company licensed the Gaucher program from, and will continue to collaborate with, Stefan Karlsson, MD, PhD, Professor of Molecular Medicine and Gene Therapy at Lund University in Sweden. Dr. Karlsson is widely recognized for his significant contributions to gene therapy and hematopoietic stem cell biology. Dr. Karlsson previously served as Chief of the Molecular and Medical Genetics Section, NIH, NNDS from 1988-1996, and as President of The International Society for Experimental Hematology. Dr. Karlsson is a frequent speaker at meetings on Gaucher disease.

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 programs in Fabry disease and acute myeloid leukemia (AML), while actively building a pipeline of therapies to treat Gaucher disease and other rare diseases. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, email us at info@avrobio.com.

Media Inquiries:
Kirsten Dupuis

Investors & Media Sign up for
Email Alerts

We’ll keep you connected to the latest news about AVROBIO’s research and pipeline.