CEO Ed Kaye was once quoted "Sarepta will own DMD" - that quote appears to be coming true… For those that are interested in potential better options, there has been some public chatter that a next generation PPMO will be coming from Sarepta, maybe as soon as next year.
…and on a Sarepta Therapeutics Message Board in May
PPMO + ... The next generation that makes the platform valuable.
Once eteplirsen validates the PMO platform, the rest of the story will start to unfold. PPMO + should reduce dosages and increase effect for the platform.
In 2009, Sarepta initiated preclinical development of a PPMO for DMD (AVI-5038) in DMD patients amenable to exon 50-skipping. As reported in Sarepta’s last Annual Report, toxicity issues with this PPMO were never successfully resolved.
...we noted unexpected toxicology findings in the kidney as part of our series of preclinical studies for AVI-5038, our PMO-based candidate designed for the treatment of individuals with DMD who have an error in the gene coding for dystrophin that can be treated by skipping exon 50. We have conducted additional preclinical studies and have not alleviated the toxicity problem. Pursuant to the terms of our agreement with Charley’s Fund, the receipt of additional funds is tied to the satisfaction of certain clinical milestones. Because of the toxicity issues with AVI-5038, satisfaction of the additional milestones under the agreement is unlikely and we do not expect to receive any additional funds from Charley’s Fund.
Despite the failure of this PPMO candidate, if the “chatter” is to be believed, Sarepta hasn’t given up on its quest to develop a PPMO for DMD. Seemingly, Murdoch University isn’t involved in that particular quest. The 4 year collaborative research agreement established last October between Sarepta and Murdoch University, which gives Professor Fletcher and Wilton’s laboratory access to Sarepta’s PMO technology, is said to be to explore the applicability of Sarepta’s PMO technology technology “for disease targets beyond Duchenne muscular dystrophy (DMD)…”
However, there are certainly some interesting overlaps.
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