Bioregnum, Gene Therapy, Insiders

Another player in the gene therapy field is gunning for the Duchenne MD crown — and here’s what they’re betting on

Most of you gene therapy enthusiasts reading this will already know about one program out of Nationwide Children’s Hospital that’s attracted considerable attention in the industry. It’s the micro-dystrophin one at Sarepta, which has snagged a big spotlight for the early — and very exciting — human data that they’ve been rolling out.

Today, a different biotech company you most likely haven’t heard nearly as much about is rolling out a new gene therapy program for Duchenne muscular dystrophy — soon to be tested in humans — that they believe can leapfrog Sarepta straight to patients.

“It’s safer and more effective compared to other approaches, including micro-dystrophin.”

That’s the CEO of Audentes talking. Matt Patterson. He’s the company co-founder who counted himself as employee #1 for the first 6 months, now with a staff of 230 and a market cap of $1.7 billion. Audentes has some early data due out on their lead gene therapy program for AT132 in a few weeks.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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