Gene therapy in the time of COVID

In recent months, biotech companies have heroically put resources and talent towards fighting COVID-19. However, we can’t ignore the many other diseases that affect people every day. BIO President and CEO Dr. Michelle McMurry-Heath recently visited a BIO member (with a mask!) that’s been continuing to work on treatments for rare diseases even as the pandemic rages on.

Meet REGENXBIO. Based just outside Washington in Rockville, Maryland, this innovative biotech is working to bring gene therapies to the market to reverse, improve, or even stop the course of a rare disease.

To adapt to the COVID environment, REGENXBIO established best practices regarding safety so lab employees and those involved in clinical studies continue to work onsite. For example, the company has imposed mandatory mask-wearing, social distancing, and plenty of hand sanitizer. 

But the company has not lost sight of its overall mission: to develop and deliver treatments to patients.

During the pandemic, REGENXBIO completed dosing of three patients in its phase 1/2 study of gene therapy for Mucopolysaccharoidosis Type II. And, after reporting positive data for an antibody gene therapy treatment in April, the company expects to continue trials in 2020. 

And there’s more success to come. REGENXBIO is 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. 

Watch this quick video with highlights from Dr. Michelle's visit:

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BIO member SAB Biotherapeutics has a big announcement regarding the clinical trial of their polyclonal antibodies to treat COVID-19—another step forward in the fight against the coronavirus.

The background: SAB is a clinical-stage biopharma based in Sioux Falls, South Dakota, using genetic engineering and antibody science to breed cows with fully human antibodies for SARS-CoV-2.  

Why cows? These large animals have robust immune systems and can produce more antibodies than a lot of animals used in biotech—and a LOT more than convalescent human patients.

Now, SAB has announced that they’ve dosed the first participant in the phase 1 clinical trial to evaluate the safety of the antibody therapeutic, SAB-185. They’re getting ready to launch phase 1b soon, too.

What makes it different: SAB’s technology “leverages the native human immune response, offering a differentiated potential high-potency therapeutic option, from conventional monoclonal and monoclonal cocktail approaches, in addressing mutating targets as recently published in Frontiers in Microbiology,” the company said. 

Why it matters: The treatment “could have significant value to protect the military, front-line responders, mission-critical staff, and high-risk populations against COVID-19,” they said. 

What they’re saying: “[P]reclinical data…demonstrate that our novel immunotherapy platform can successfully neutralize mutated strains of viruses,” said SAB’s Co-Founder and CEO Eddie J. Sullivan. “Further, SAB-185 preclinical data has demonstrated highly-potent neutralizing antibodies to SARS-CoV-2 that are more potent than human convalescent plasma serum samples available against the virus. Taken together, these data suggest that SAB-185 may be effective even as SARS-CoV-2 continues to evolve.”