It wasn’t long ago that “gene therapy treatment” sounded like science fiction, but it has now become a reality and cutting-edge work in the field is being carried out in Montgomery County, Maryland, by REGENXBIO, a leading clinical-stage biotechnology company that seeks to improve lives through the curative potential of gene therapy.
“Our products are designed as medicines that deliver genes to cells to address genetic defects or to enable cells to produce therapeutic proteins in the body that are intended to impact underlying diseases,” explained Ken Mills, the President and CEO of REGENXBIO.
The company’s products in clinical development are meant to provide long-lasting effects, potentially altering the course of a disease.
REGENXBIO is currently developing gene therapy products for the treatment of retinal, metabolic and neurodegenerative diseases.
“In some cases, the therapy is delivered through a simple intravenous infusion, maybe for a few hours,” Mills explained. “Patients enter the hospital, receive the infusion and they’re usually able to go home that day while the gene therapy is taking effect.”
In other cases, involving the treatment of eye diseases, for example, a doctor may need to use a needle to make an injection to help cells in the eye to generate the targeted therapy needed to impact disease.
“It can vary, but the key element of gene therapy that’s really special is these are meant to be one-time treatments, no matter how they’re introduced into the body,” Mills said.
How does it work?
Most traditional medical treatments that patients receive are designed to treat only the symptoms of a disease rather than the underlying cause.
Gene therapy treatment, on the other hand, literally treats a disease from the ground up.
“What we find is that many diseases happen at the DNA level,” Mills explained. “When we identify that something’s wrong with DNA, with gene therapy we can create a new piece of DNA and put it into the body.”
That new DNA copy helps to replicate what should have been happening in the body to begin with, producing something that wasn’t there before or providing more structural stability to cells or tissues.
“It’s starting to become a technology and a class of medicine that’s used more broadly,” said Mills, who pointed specifically to Zolgensma, a gene therapy treatment for infants and toddlers who suffer from spinal muscular atrophy.
In 2019, Zolgensma became one of the first gene therapy products to be approved by the U.S. Food and Drug Administration. Zolgensma is built on REGENXBIO’s NAV Technology platform.
Spinal muscular atrophy, which is often fatal, causes children to have problems holding their head up, swallowing and breathing.
“Zolgensma is a treatment that has allowed children to continue with developmental milestones and live normal lives,” said Mills. “It’s really a form of a miracle, and our science is a major contributor to that.”
New facility in Rockville
REGENXBIO is rapidly expanding in Montgomery County, recently opening a state-of-the-art facility in Rockville that will help the company continue its research and clinical trials.
It is one of the few gene therapy manufacturing facilities in the United States.
“The REGENXBIO Manufacturing Innovation Center isa leading state-of-the-art manufacturing facility that will support new studies as well as the advancement of new commercial treatments,” Mills said. “It is a facility that we expect will manufacture gene therapies in order to deliver them throughout the world.”
Mills said that the company chose Montgomery County due to its “diverse atmosphere,” including the fact that it’s home to both the Food and Drug Administration (FDA) and the National Institutes of Health (NIH).
“Montgomery County is a perfect place for a biotech company to grow,” Mills said, adding that the Montgomery County Economic Development Corporation has been “an essential partner” for his team.
The MCEDC is a public-private partnership that collaborates with county and state officials to help connect businesses to resources, site selection, funding, incentives and permitting, among other things.
“They have been a great resource,” Mills said. “The Montgomery County Economic Development Corporation is constantly making connections for us and asking how they can support our ideas and logistics.”
REGENXBIO went from having fewer than 30 employees in 2015 to employing about 400 people now.
The company’s goal is to have at least five of its medicines in late-stage development or approved by the FDA by 2025.
“The next two or three years are going to be a an incredibly busy time for us,” Mills said. “The people at REGENXBIO – we are really committed to the mission of bringing medicines, ourselves, to patients.”
Montgomery County Economic Development Corporation (MCEDC): Maryland’s Montgomery County: The Immunology Capital Next to the Nation’s Capital. Home to NIH, FDA, top talent, partnerships and life science success for you.