Avrobio Pauses Dosing in Phase 1/2 Trial of Gene Therapy for Gaucher

Avrobio Pauses Dosing in Phase 1/2 Trial of Gene Therapy for Gaucher
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Due to the current COVID-19 outbreak, Avrobio announced that it has paused dosing new patients in clinical trials of its gene therapy candidates, including a Phase 1/2 study testing AVR-RD-02 in people with Gaucher disease type 1.

This trial’s first Gaucher patient is enrolled and has undergone a blood draw. The company expects to treat this person before or by June, but the trial site has final say. Dosing in other patients may begin in the second half of this year, Avrobio says.

Avrobio is moving forward with identifying patients interested in taking part in all of its ongoing trials. Its manufacturing activities are not affected by the pandemic.

“Our first priority during this unprecedented time is the health of our employees, our patients and their communities, and the employees of our clinical sites, partners and vendors,” said Geoff MacKay, Avrobio’s president and CEO, in a press release.

“We continue to support patient identification efforts across our clinical trials in Canada, Australia, and the United States,” MacKay added. “As the global healthcare community responds to the increase in COVID-19 cases, many hospitals, including our clinical sites, have temporarily paused elective medical procedures, which includes dosing of new patients in clinical trials.”

Avrobio has moved its operations mostly online or virtual interaction, in keeping with recommendations by health authorities. An exception has been made for essential laboratory work, which is being conducted under additional safety measures.

MacKay added that his company is continuing to monitor “this rapidly evolving situation” for its potential impact on both our clinical trial programs and business generally.”

People with Gaucher have a faulty GBA gene, which affects the activity of the enzyme beta-glucocerebrosidase (GCase) and leads to an accumulation of a lipid (fat) called glucocerebroside in cells.

AVR-RD-02 is designed to slow, or even reverse, disease progression, including GBA-related Parkinson’s disease, which is common in GD type 1 patients. It uses Avrobio’s plato platform and a harmless lentiviral vector to deliver a functional copy of GBA to the patients’ hematopoietic stem cells (blood cell precursors), cultured in a lab dish. The modified cells are then reintroduced to patients, with the aim of restoring the production and activity of GCase.

The Phase 1/2 trial (NCT04145037), called GAU-201, is studying the safety and efficacy of AVR-RD-02 in eight to 16 patients ages 16 to 35, including those who have not had any prior treatment and those on enzyme replacement therapy.

The U.S. Food and Drug Administration (FDA) designated AVR‑RD‑02 an orphan drug in 2019, qualifying Avrobio for several incentives intended to support clinical trials of AVR‑RD‑02 and, if approved, the therapy’s commercialization.

In addition to Gaucher, the company has gene therapy trial programs underway in Fabry disease and cystinosis. Similar to the study in Gaucher, patient identification continues in these two programs. Data collection is also expected to continue, although timing could be affected.

Avrobio created an internal COVID-19 response team last month to monitor and recommend responses to the evolving situation. The company expects to provide a further update next month. It does not expect the outbreak to have a significant impact on regulatory, research or preclinical work.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”

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José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”

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