AMD is all about different genes.- MDA

New Research leading to clinical trials

Rockville gene therapy company to begin trials for treatment for leading cause of blindness

by: Tina Reed

Rockville-based RegenxBio Inc. has received Food and Drug Administration approval to begin clinical trials of its gene therapy to treat wet age-related macular degeneration, a leading cause of blindness.

RegenxBio’s gene therapy technology introduces healthy genes into patients suffering from genetic disorders, often rare ones affecting the central nervous system and eyes. The technology replaces the DNA in a type of virus that does not cause illness with a specific gene in order to deliver gene therapy to humans. The technology was originally developed at the University of Pennsylvania.

With the approval, RegenxBio (NASDAQ: RGNX) has moved its focus from using gene therapy for rare diseases into a mainstream market, said CEO and President Ken Mills. Another of RegenxBio’s gene therapy drug candidates is a treatment for a rare neurodegenerative disease in children called mucopolysaccharidosis Type I, which impacts about 1,000 patients a year.

“This shows the breadth and depth of what RegenxBio is doing as a global company to address multiple diseases for different populations,” Mills said. Mills previously said the company has followed a strategy of targeting diseases where there are no treatments and the fix involves a change to a single gene.

Often patients who receive treatment for wet age-related macular degeneration must receive injections into the eye multiple times a month, Mills said. “We’ve talked to a lot of people about it and they get anxious about it,” Mills said. “Then patients don’t go to their appointment and then it gets worse.”

The gene therapy, which still faces years of clinical testing, would allow patients to receive treatment with a one-time injection, he said. The company expects to begin trial enrollment by mid-2017….

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Source: Washington Business Journal