AVROBIO Obtains $60 Million for Phase 2 Trial of Its Fabry Disease Gene Therapy

AVROBIO Obtains $60 Million for Phase 2 Trial of Its Fabry Disease Gene Therapy

AVROBIO has obtained $60 million in additional financing to start a Phase 2 clinical trial of its gene therapy for Fabry disease.

The second round of financing will also help the company continue developing treatments for other lysosomal storage disorders, Gaucher disease, cystinosis, and Pompe disease.

A Phase 1 trial (NCT02800070) of the Favry therapy, AVR-RD-01, led to a surge in the production of a protein that is deficient in people with the disease, according to six-month results covering one patient.

In fact, the treatment led to alpha-galactosidase A protein levels reaching those in people without the disease, researchers said. A mutation in the GLA gene causes the protein shortage.

Researchers continue to recruit patients for the Phase 1 trial in locations across Canada.

Cormorant Asset Management and Surveyor Capital led the new financing round. Atlas Venture, SV Health Investors and Clarus Ventures, which participated in the first round, took part in the second as well. Other second-round participants included Aisling, Brace Pharma Capital, Eventide Asset Management and Morningside. Atlas Venture, SV Health Investors and Clarus Ventures.

“Gene therapy is a class of medicines that has made breakthroughs in the biotechnology landscape, and AVROBIO has carved out a strong position with its lentiviral platform focused on the significant therapeutic and market opportunities for gene therapies for lysosomal storage diseases,” Bruce Booth, an Atlas Venture partner who is chairman of AVROBIO’s board, said in a press release.

“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,” Booth said. “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.”

AVROBIO said it expects its single-dose Fabry therapy to be long-lasting. In fact, it could be a life-long cure for some patients, it said.

The therapy involves replacing the GLA gene that’s faulty in Fabry with a healthy one that can generate normal α-galactosidase protein levels.

AVROBIO developed a proprietary third-generation virus delivery system to get its gene therapies to where they need to go in the body.

“We are delighted by this exceptional group of investors supporting our plans to build AVROBIO as a leader in the field of lentiviral gene therapy,” said Geoff MacKay, the president and CEO of AVROBIO.

“We are excited to continue to rapidly advance our clinical [trial] program for AVR-RD-01 in Fabry disease, while also moving two additional gene therapies from our pipeline into clinical trials,” he said. “We are on the leading edge of applying gene therapy as a potential single-dose curative treatment for lysosomal storage disorders, and our vision is to use our lentiviral platform to have broader impact for patients by shifting the paradigm for a wide range of other diseases.”

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