Sarepta Therapeutics CEO has a 'big ambition' for muscular dystrophy gene therapy

  • Sarepta Therapeutics CEO Doug Ingram wants the company to start giving its Duchenne muscular dystrophy gene therapy to children in the approval trial by the end of this year.
  • "We have a big ambition to potentially be to the community within two years thereafter," he says.

Sarepta Therapeutics CEO Doug Ingram told CNBC on Friday he has a "big ambition" to get the company's Duchenne muscular dystrophy gene therapy to patients as quickly as possible.

He wants Sarepta to be in a position to start dosing children in the approval trial by the end of this year.

"We have a big ambition to potentially be to the community within two years thereafter," Ingram said in an interview with CNBC's Meg Tirrell on "Closing Bell."

Duchenne muscular dystrophy is a rare genetic disease that causes muscle wasting and can be fatal before patients turn 30. Symptoms usually start in early childhood, usually between 3 and 5 years old. It primarily affects boys but in rare cases can affect girls.

The biopharmaceutical company saw positive preliminary results in a phase 1/2a clinical trial, which sent shares soaring. However, in July, the Food and Drug Administration placed the trial on clinical hold because of a trace amount of DNA fragment found in research-grade third-party supplied plasmid.

"We've had extraordinary early results," Ingram said, but noted that more children need to be treated. The preliminary results were from three children who received the gene therapy.

Ingram said the FDA hold occurred, in part, because of a "manufacturing issue with a supplier" and that Sarepta has "fully responded" to the FDA.

"Our goal and belief is it will be off clinical hold very soon and it shouldn't affect the timing of the things we're going to do," he said.

Ingram hopes to get in front of the FDA in the next 60 days or so to talk about Sarepta's clinical program and the path to get the treatment to the community.

"We are in the lead, but there are other credible programs behind us. And if we can't work fast with a sense of urgency, someone else would do it," he said.

"Our goal is to frankly be fueled by competition to hopefully ensure that we have the most elegant program, we're in the lead and that we can get to this community as fast as possible."

Shares of Sarepta Therapeutics are up more than 200 percent in the last 12 months.

— CNBC's Angelica LaVito contributed to this report.