More tests needed to develop a gene therapy for a rare disease: CEO

Sarepta Therapeutics CEO Douglas Ingram told CNBC of which more tests are needed to develop a gene therapy to treat a rare genetic disease.

On Tuesday, the medical research along with drug development company released data on an experimental treatment for patients with Duchenne muscular dystrophy, or DMD.

“This kind of can be preliminary results,” Ingram said on “Closing Bell” on Tuesday. “We need to be careful.”

The successful preliminary trial sent shares of Sarepta Therapeutics soaring along with opened the door for further testing.

The initial tests, on just three children during a three-month period, are “potentially transformative,” Ingram said, however he acknowledged of which more research needs to be done.

“We need to treat more kids,” he said. “We’re going to treat 12 kids versus 12 placebo kids. along with we need to watch them for some time, perhaps a year.”

“however we don’t want to wait of which long,” he said. “Because every single day of which we delay, these kids are being damaged. People have been trying to find a solution to This kind of for decades.”

A lack of dystrophin, a protein of which helps keep muscle cells intact, causes the disorder along with results in muscle degeneration along with weakness. of which affects boys predominantly. About 16 out of every 100,000 people contain the disease, according to the Centers for Disease Control along with Prevention.

People with the disease usually require a wheelchair by around age 11 along with have a life expectancy of 20 to 25 years old, Ingram said.

“These kids have muscle damage every time they move,” he said.

In damaged muscles the enzyme creatine kinase can leak into the bloodstream. Large amounts of the enzyme can signal Duchenne muscular dystrophy.

inside preliminary tests, patients were given a gene therapy of which acted as a substitute for the dystrophin.

The results of the trial showed a nearly 0 percent average reduction of creatine kinase levels after using the company’s drug treatment.

The company’s shares surged more than 50 percent Tuesday after the results of the trial were announced, along which has a day after the Food along with Drug Administration took the company’s program off clinical hold. Sarepta Therapeutics’ stock closed up more than 36 percent Tuesday.

Ingram did not comment on the potential cost of the drug. He said if results of larger tests are also positive, then the company can seek FDA approval.

“Our goal, eventually, can be to get This kind of therapy to all patients,” Ingram said. “of which means we have to finish the clinical trial.”

— CNBC’s Angelica LaVito contributed to This kind of report.

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