Cutting-Edge Gene Therapy Treatments Being Developed in Montgomery County, Maryland

June 9, 2022, 3:04 PM | by Nick Iannelli

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.”

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