Isis Pharmaceuticals, Inc. Message Board

This is just a scratch of the future ....any real good Big Pharma could see that.

FDA ,Sarepta drug production meeting....

30 days of receiving the white paper package..........The FDA is far more better than it has ever been.

Sarepta Therapeutics Initiates Dosing in Phase I Multiple Ascending Dose Study of Drug for Treatment of Marburg Virus Full Text
Sarepta Therapeutics, 05/20/2013

Sarepta Therapeutics, Inc., a developer of innovative RNA–based therapeutics, announced that it has initiated dosing in a Phase 1 multiple ascending dose (MAD) clinical trial of AVI–7288, the Company's lead drug candidate for the treatment of Marburg virus infection. The Phase I MAD study is designed to characterize the safety, tolerability and pharmacokinetics of AVI–7288 after repeat dosing in healthy adult volunteers. The initiation of this study follows the successfully completed Phase 1 single ascending dose study, which showed AVI–7288 was well tolerated in healthy volunteers. Sarepta is developing AVI–7288 under a contract from the U.S. Department of Defense through the Joint Project Manager Transformational Medical Technologies (JPM–TMT) Project Management Office. AVI–7288 utilizes Sarepta's advanced and proprietary PMOplus® chemistry.

see ..............
US2012/046783
Publication Date:
January 24, 2013
Filing Date:
July 13, 2012

Assignee:
SAREPTA THERAPEUTICS, INC. (3450 Monte Villa Parkway, Suite 101Bothell, Washington, 98021, US)
IVERSEN, Patrick, L. (5902 Northwest Fair Oaks Place, Corvallis, Oregon, 97330, US)

I would guess..................................................................................

Today may 17 2013 at 5.30pm......................................................................................................................................see poster abstract i posted early today.....

Sarepta Therapeutics Inc’s (SRPT) Technology Shows Dramatic Results as DMD Patients Regain Functions
May 17, 2013

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.”