ONE PIPELINE for a better future
We are building a pipeline of compelling investigational therapies in indications where we believe gene therapy can have the greatest impact for people living with genetic disorders.
Our investigational therapies are being evaluated in clinical studies in Gaucher disease type 1 and type 3 and Hunter syndrome.
Discover more about how HSC gene therapy can have a head-to-toe impact
AVROBIO is studying the safety and efficacy of its investigational gene therapy in individuals with Gaucher disease type 1 (GD1) and type 3 (GD3).
The Phase 1/2 clinical trial for GD1 (named Guard1) is enrolling in the U.S. and Canada. The trial is intended to recruit 8 to 16 individuals between the ages of 16 and 50 with GD1, including both those who are treatment-naïve and those who are stable on enzyme replacement therapy.
AVROBIO plans to initiate a global registrational Phase 2/3 trial for GD3, (named Guard3) in the second half of 2023, subject to regulatory alignment.
AVROBIO’s investigational gene therapy for neuronopathic mucopolysaccharidosis type II (nMPSII or Hunter syndrome), is being studied in a Phase 1/2 clinical trial sponsored by the University of Manchester, U.K. The 24-month, single-arm, open label study will evaluate the gene therapy’s safety and tolerability, as well as its pharmacodynamic and clinical efficacy. The trial aims to recruit up to five patients with nMPS II who are aged between 3 and 12 months.
AVROBIO has a preclinical research program for a gene therapy for Pompe disease. We have shown a favorable preclinical safety and efficacy profile in a mouse model of Pompe disease.
Treating the Whole Body
Read about our personalized conditioning regimen
Read about our mission and vision.
Learn more about our personalized approach to gene therapy.
CloseToday is MPS Awareness Day (MPS II is also known as Hunter syndrome) and we want to recognize the incredible work that patient advocacy groups do year-round to support the Hunter syndrome community.
Learn more about Hunter syndrome and the patient advocacy groups’ efforts here: