Friday, November 28, 2014
BioMarin $700M Acquistion of Prosensa Comes Down to Attraction of RNA Therapeutics
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Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
In this study, as a proof-of-concept of such gene therapy for DMD, we performed and demonstrated genetic correction of the dystrophin gene in patient-derived iPSCs by using three different methods: (1) disruption of the splicing acceptor to skip exon 45, (2) introduction of small indels to modulate the protein reading frame, and (3) knockin of the missing exon 44 to restore the full protein coding region. We then performed comprehensive genome-wide mutation analyses to assess the risk of off-target mutagenesis in 14 iPSC clones treated according to the TALEN or CRISPR approach. Our results demonstrate that genetic correction by these approaches in patient-derived iPSCs considerably lowers the risk of off-target mutagenesis and thus holds promise for DMD gene therapy.
http://www.cell.com/stem-cell-reports/fulltext/S2213-6711%2814%2900335-X
Failing to Rise to the Challenge What the FDA should learn from Ebola. Dec 1, 2014, Vol. 20, No. 12
A recent example of the FDA’s indefensible inconsistencies is its October decision to demand more data yet again—without clear, science-based standards—for companies seeking approval for several new drugs that should be available now to patients with the fatal disease of Duchenne muscular dystrophy.
What makes the muscular dystrophy pronouncements worse is that there are signs that they are driven by the FDA’s worst cultural tendencies: retaliation behind closed doors, “leveling the playing field” for competing companies by slowing down the company in the lead, and improperly relying on outside advisers with a strong financial interest in the outcome of its decisions.
As the new Congress establishes its oversight priorities, this shameful mess should be high on its list.
The FDA should learn from its mistakes. It needs to be more proactive with guidance on the standards for approval of drugs for Ebola and other tropical diseases, more transparent about the safety and efficacy of investigational drugs for Ebola, and more understanding about why randomized trials for rare diseases are often not feasible. When it comes to children dying of muscular dystrophy, the FDA needs to show more of the urgency it displayed when approving investigational Ebola drugs for heroic public health workers.
Michael Astrue is a former commissioner of Social Security and a former HHS general counsel.
http://www.weeklystandard.com/articles/failing-ris-challenge_819713.html?page=2
Imagine how wealthy the founders of Voyager will be when they receive an offer like that. Dirk and Kay will be chuffed.
Especially when they didn't even have to take a licence from their nemesis, BLT.
Though there is a small chance that it could end up being Avocell MkII.
Where is PFE?
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