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Prosensa begins FDA submission process for DMD drug
Authored by James Sheppard
Massachusetts biotech continues to excite with new investments
Authored by Karl Simpson
Posted in M&A Finance and Funding, Orphan Drugs and Rare Diseases, Pharmaceutical business
Tagged Abingworth Management, BioPharma America, Biotechnology cluster, Boston, Brookside Capital, Cristina Csimma, Cydan, David Mott, Deerfield, DELPHI, Dicer Substrate siRNA, Dicerna Pharmaceuticals, drug delivery technologies, Duchenne Muscular Dystrophy, EDI200, Edimer Pharmaceuticals, EnCore™, exon skipping, Exportin-1, Forsite Capital Management, Genzyme, Inhibitors of Nuclear Export, innovation, investors, Karyopharm Therapeutics, Massachusetts, Neil Kirby, New Enterprise Associates, New Leaf Venture Partners, NEZ, Omega Funds, oncology market, Orphan Drug, Prosensa, RA Capital, Rare Disease, Sanofi, Series B, SR One, Third Rock Ventures, TKT, undruggable disease, Vertex Pharmaceuticals, X-linked Hypohidrotic Ectodermal Dysplasia, XLHED
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Sarepta’s hopes high for first Duchenne (DMD) therapy
Authored by James Sheppard
Posted in Orphan Drugs and Rare Diseases
Tagged AVI Biopharma, AVI-7100, Department of Defense, DMD, Duchenne Muscular Dystrophy, Ebola, EMEA, Eteplirsen, exon skipping, FDA, Infectious Disease, Influenza, Liftstream Rare Disease Day, Marburg Virus, NIH Clinical Center in Bethesda, Phase II Trials, PMO Chemistry, Rare Disease Day 2013, RDD13, Sarepta Therapeutics, UCL, University College London, USA
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