Gene Therapy in the Midst of COVID

During the last several months, biotech companies have heroically trained their resources and talent toward fighting the global COVID pandemic. The entire world recognizes that it is science that ultimately will help us get back to our former life and help curtail deaths, sickness and economic hardship. At the same time, we cannot ignore that other diseases still affect people every day.

Thankfully, companies like REGENXBIO have continued to work on treatments for rare diseases, even while the pandemic rages on. Focusing on groundbreaking gene therapy technology, the company’s mission is to bring medicines to the market that will reverse, improve or stop the course of a rare disease.  

BIO’s President and CEO, Dr. Michelle McMurry-Heath recently toured REGENXBIO lab and facility in Rockville, MD and talked with the team about the important work the company is doing despite the ongoing pandemic.

“It’s so important for non-COVID based biotechnology companies to keep on working. The patients don’t stop waiting just because of COVID. COVID is, of course, an incredible concern, but all of the illnesses are still out there and still need cures and solutions. We are gratified that our companies are dual tracking and really trying to do both…”

To adapt to the COVID environment REGENXBIO established best practices regarding safety such as sending some people home to work, but it did not lose sight of its overall mission to develop and deliver treatments to patients. Laboratory employees and others involved in clinical studies continue to work on the premises so the work towards delivering transformative and long-lasting treatments can continue. For those onsite, the company has imposed strict safety guidelines such as mandatory mask-wearing, social distancing and plenty of hand sanitizer.

During the pandemic, REGENXBIO completed dosing of three patients in its Phase I/II study of gene therapy for Mucopolysaccharoidosis Type II. And after reporting positive data for an antibody gene therapy treatment in Wet AMD in April, the company expects to pursue continued trials of this candidate in 2020, using various delivery methods and adding studies of diabetic retinopathy. REGENXBIO is also on track to open a new manufacturing facility and expects to make further progress in other programs across its pipeline during the second half of this year.

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