AVROBIO

Gene therapy targets diseases caused by genetic mutations beyond standard care.

Lysosomal disorders are rare diseases caused by gene mutations that impair lysosome function, leading to toxic material buildup and tissue damage.

AVROBIO aims to halt, reverse, or prevent lysosomal disorders with a single gene therapy dose.

AVROBIO is developing gene therapies for genetic disorders where the approach may have significant impact.

Gaucher disease is a rare genetic disorder caused by mutations in the GBA gene, which reduces production of the enzyme glucocerebrosidase (GCase). This deficiency leads to toxic buildup of fatty substances like glucosylceramide and glucosylsphingosine in organs such as the spleen, liver, and bone.

AVROBIO’s leadership team discusses business practices, including pipeline strategy and culture building.

The company’s team advances gene therapy research and shares findings with the global gene therapy community.

AVROBIO focuses on delivering gene therapies for genetic diseases through collaboration and inclusivity.

ONE TEAM striving to pioneer a new frontier in medicine

ONE COMPANY with a vision to bring personalized gene therapy to the world.

For general questions: [email protected]

AVROBIO’s gene therapy aims to halt or reverse disease progression, reduce symptoms, and eliminate chronic treatment burdens.

AVROBIO develops gene therapies for rare diseases by collaborating with researchers, patient groups, physicians, and regulatory agencies.

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AVROBIO adds Annalisa Jenkins to its board, leveraging her gene therapy and pharmaceutical leadership. Jenkins previously served as CEO of Dimension Therapeutics, Global R&D Head at Merck Serono, and Global Medical Affairs Head at Bristol-Myers Squibb. The biotech firm focuses on developing gene therapies for rare diseases.

Apr 2018AVROBIONews article

AVROBIO secures $60 million in Series B funding to advance its gene therapy pipeline targeting lysosomal storage disorders. The company plans to initiate a Phase 2 trial for its lead therapy, **AVR-RD-01**, which shows early promise in treating **Fabry disease**. The financing supports a multi-product portfolio, including therapies for **Gaucher disease, cystinosis, and Pompe disease**, leveraging its lentiviral gene delivery platform for broader genetic disease applications.

Feb 2018AVROBIONews article

AVROBIO’s first patient with Fabry disease achieved normal plasma α-galactosidase A enzyme activity within 45 days after a single dose of its lentiviral gene therapy, AVR-RD-01. The patient maintains normal enzyme levels six months post-treatment. Data were presented at the 59th Annual Meeting of the Japanese Society for Inherited Metabolic Diseases in October 2017.

Oct 2017AVROBIONews article

AVROBIO adds a gene therapy program for cystinosis, its fourth initiative targeting lysosomal storage disorders (LSDs). The company specializes in developing gene therapies for rare diseases, aiming to deliver transformative treatments. This expansion strengthens its pipeline in the LSD space, where it already has active programs.

Oct 2017AVROBIONews article