Sarepta Therapeutics Inc’s (SRPT) innovative technology treating gene mutations in Duchenne muscular dystrophy patients is showing remarkable results in clinical trials, and this technology has the potential to treat other gene mutations within DMD as well as other diseases, making SRPT a promising big story in corrective medicine, says Finny Kuruvilla, Portfolio Manager of the Eventide Gilead Fund.
“One of the things that’s really interesting about this company that I don’t think the market has fully digested is the fact that the way they treat Duchenne muscular dystrophy…DMD patients have a mutation in a gene called dystrophin, and their muscle cells tend to rupture and lyse… [Sarepta‘s technology] enables the patient to be able to make normal dystrophin like you and I have, unlike what they’ve had before. It’s nearly science fiction; real mind-blowing technology,” Kuruvilla said.
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SRPT‘s treatment has already shown dramatic effects in a handful of patients that are showing incredible turnaround and a regain of function that was not possible before, Kuruvilla says. He adds that the technology is showing promise in being generalizable to other areas of DMD as well as in different diseases, placing SRPT at the forefront of one of biotech’s potentially historic breakthroughs.
“Right now, I think the market is incompletely valuing it on its lead indication, this 15% of DMD subset, but if you begin to think about, and have a little bit of imagination about, other forms of Duchenne muscular dystrophy and other diseases, this becomes one of the most significant technology platforms that biotech has seen in a number of years,” Kuruvilla said. “This can be one of the big stories not just in DMD, but in corrective medicine and truly disease-modifying therapy to come around in a long time.”
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