Sarepta CEO has big ambition for muscular dystrophy gene therapy

Sarepta Therapeutics CEO Doug Ingram told CNBC on Friday he includes 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 from the approval trial by the end of This particular 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 can be a rare genetic disease in which causes muscle wasting in addition to can be fatal before patients turn 30. Symptoms usually start in early childhood, usually between 3 in addition to 5 years old. This particular primarily affects boys however 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 in addition to 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, however noted in which more children need to be treated. The preliminary results were by three children who received the gene therapy.

Ingram said the FDA hold occurred, in part, because of a “manufacturing issue that has a supplier” in addition to in which Sarepta has “fully responded” to the FDA.

“Our goal in addition to belief can be This particular will be off clinical hold very soon in addition to This particular shouldn’t affect the timing of the things we’re going to do,” he said.

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

“We are from the lead, however there are different credible programs behind us. in addition to if we can’t work fast that has a sense of urgency, someone else would likely do This particular,” he said.

“Our goal can be to frankly be fueled by competition to hopefully ensure in which we develop the most elegant program, we’re from the lead in addition to in which we can get to This particular community as fast as possible.”

Shares of Sarepta Therapeutics are up more than 0 percent from the last 12 months.

— CNBC’s Angelica LaVito contributed to This particular report.

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