Our 
Pipeline

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 Fabry disease, Gaucher disease and cystinosis. 

Investigational Gene Therapy

  • Proof-of-concept
  • IND-Enabling
  • Phase 1/2
  • Proof-of-concept
  • IND-Enabling
  • Phase 1/2

Fabry AVR-RD-01

AVR-RD-01

AVROBIO's investigational gene therapy for Fabry disease is currently being studied in two clinical trials.

An investigator-sponsored Phase 1 trial in Canada is fully enrolled, with five patients dosed.

FAB-201, the Phase 2 trial, has dosed four treatment-naïve patients through December 2019 and continues to enroll in Australia, the U.S. and Canada.

Learn more about the FAB-201 trial here.

  • Proof-of-concept
  • IND-Enabling
  • Phase 1/2

Cystinosis AVR-RD-04

AVR-RD-04

AVROBIO's investigational gene therapy for cystinosis is being studied in a Phase 1/2 investigational trial sponsored by the University of California, San Diego. The first patient was dosed in October 2019. The single-arm trial is expected to enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis.

For more information, click here.

  • Proof-of-concept
  • IND-Enabling
  • Phase 1/2

Gaucher AVR-RD-02

AVR-RD-02

AVROBIO's investigational gene therapy for Gaucher disease is being studied in a Phase 1/2 clinical trial to evaluate the safety and efficacy in individuals with Gaucher disease type 1.

The trial is now enrolling in Canada and Australia and AVROBIO plans to open clinical sites in the U.S. The trial is intended to recruit 8 to 16 individuals between the ages of 16 and 35 with Gaucher disease type 1, including both those who are treatment-naïve and those who are stable on enzyme replacement therapy.

For more information, click here.

  • Proof-of-concept
  • IND-Enabling
  • Phase 1/2

Hunter syndrome AVR-RD-05

AVR-RD-05

AVROBIO has a preclinical research program for mucopolysaccharidosis type II (MPS II), or Hunter syndrome. The program is being developed in collaboration with Brian Bigger, Ph.D., who has published favorable preclinical data in a mouse model of Hunter syndrome.

  • Proof-of-concept
  • IND-Enabling
  • Phase 1/2

Pompe AVR-RD-03

AVR-RD-03

AVROBIO has a preclinical research program for an investigational 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.

AVROBIO

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About us

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Our approach

Learn more about our personalized approach to gene therapy.