Avrobio pulls in $60M to push Fabry, Gaucher candidates through the clinic

Illustration of three DNA helices
Avrobio's Fabry treatment involves removing a patient’s stem cells, genetically modifying them and then infusing them back into the patient. (Darwin Laganzon)

Avrobio has raised $60 million in series B financing to “aggressively” advance its gene therapy pipeline for lysosomal storage disorders, including moving its lead asset, a treatment for Fabry disease, into phase 2.

Based on its lentiviral gene therapy platform, Avrobio’s Fabry candidate, ACR-RD-01, is poised to enter phase 2 trials this year, based on promising six-month data from its phase 1 trial. The treatment involves removing a patient’s own stem cells, genetically modifying them by adding a functional copy of the defective gene and then infusing them back into the patient.

“Avrobio has made remarkable progress with its gene therapy platform, including a very compelling initial clinical response in the first patient with Fabry disease to receive the company’s lead gene therapy candidate,” said Bruce Booth, partner at Atlas Venture, which participated in the financing. “We have great confidence in the high caliber management team, and the rigorous science and clinical programs behind the gene therapies for Fabry disease and a range of other lysosomal storage disorders.”

While there already exists a treatment for Fabry—namely enzyme replacement therapy in the form of biweekly infusions—Avrobio is looking to go one better.

“What enzyme replacement therapy succeeds in doing is slowing down the decline of the disease, but the disease does continue to progress,” said CEO Geoff MacKay. The value of gene therapy lies in giving a one-time infusion of genetically modified cells that then deliver gene expression to all affected cells, tissues and organs. Not only would this free the patient from biweekly infusion for his or her entire life, but it also could stave off disease progression, he said.

The funding will also carry Avrobio’s programs in Gaucher disease and cystinosis into phase 1. The company also has a treatment for Pompe disease in the works, but it’s a little further back in development.

“We will develop a majority of the preclinical work around Pompe, but it is not intended to get into the clinic as part of this financing time frame,” said MacKay.

While the company has zeroed in on lysosomal storage disorders, it has plans to apply its gene therapy platform to other diseases.

In fact, when Avrobio raised a $25 million series A back in August 2016, it had programs in Fabry and acute myeloid leukemia. The latter appears to have been dropped—its last mention was in a March 2017 press release announcing the addition of Gaucher disease to Avrobio’s pipeline.