ONE PIPELINE for a better future

Targeting diseases where there is an urgent need for new approaches

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.

Investigational Gene Therapy

Treating the Whole Body

Discover more about how HSC gene therapy can have a head-to-toe impact

  • Preclinical
  • Early-stage clinical development
  • IND-Enabling
  • Phase 1/2

Gaucher disease AVR-RD-02

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.

For more information on Guard1, click here.


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.

For more information on Guard3, please contact medinfo@avrobio.com.

  • IND-Enabling
  • Phase 1/2

Hunter syndrome AVR-RD-05

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.

For more information on the trial, contact mpsiitrial@manchester.ac.uk.

  • IND-Enabling
  • Phase 1/2

Pompe AVR-RD-03

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.

Safety and efficacy have not been established. Our investigational therapies have not been approved by the U.S. Food and Drug Administration or any other regulatory agency.

Learn more about access to our clinical trials.

Learn more about our commitment to people living with genetic disorders.


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Treating the Whole Body

Read about our personalized conditioning regimen

About Us

Read about our mission and vision.

Our Approach

Learn more about our personalized approach to gene therapy.