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Our vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into patients’ own hematopoietic stem cells (HSCs), with the goal to durably express the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for cystinosis and Gaucher disease type 1, as well as preclinical programs for Gaucher disease type 3, Hunter syndrome and Pompe disease. Our proprietary plato® gene therapy platform is designed to be scaled to support late-stage clinical development and commercialization globally. We are headquartered in Cambridge, Mass. For additional information, visit and follow us on Twitter and LinkedIn.


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Events & Presentations

Our News Latest press releases

Sep 27, 2022

AVROBIO to Present New Preclinical Data on Vector Safety at ESGCT Annual Congress

CAMBRIDGE, Mass. --(BUSINESS WIRE)--Sep. 27, 2022-- AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that it will present new preclinical data on novel assays used to evaluate lentiviral vector

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Sep 20, 2022

AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis

Second AVROBIO gene therapy program that has been granted rare pediatric disease designation by FDA AVR-RD-04 has previously received orphan drug designation from FDA and EMA CAMBRIDGE, Mass. --(BUSINESS WIRE)--Sep. 20, 2022-- AVROBIO, Inc . (Nasdaq: AVRO), a leading clinical-stage gene therapy

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Sep 14, 2022

AVROBIO Announces Neuronopathic Mucopolysaccharidosis Type II (nMPS-II) or Hunter Syndrome Clinical Trial Application (CTA) Accepted by U.K. Regulatory Agency to Initiate Pediatric Phase 1/2 Gene Therapy Trial

First in-human pediatric Phase 1/2 study to evaluate the safety and tolerability of first-in-class gene therapy targeting infants age ≥3 months and ≤12 months, diagnosed with nMPS-II University of Manchester ( U.K. ) expects to initiate clinical trial later this year U.S.

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