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Novartis’ breakthrough therapy for a rare muscle-wasting disease
TweetSkeletal muscle On August 20, 2013, Novartis announced in a press release that the FDA had granted breakthrough therapy designation to its experimental agent BYM338 (bimagrumab) for treatment of...
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Agios Pharmaceuticals becomes a clinical-stage company!
TweetAgios Efstratios, Greece. Source: Christef http://bit.ly/HK636F In a news release on September 23, 2013, Agios Pharmaceuticals (Cambridge, MA) announced that it had initiated its first clinical...
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RNAi therapeutics stage a comeback
TweetTransthyretin protein structure Not so long ago, the once-promising field of RNA interference (RNAi)-based drugs was on the downswing. This was documented in our August 22, 2011 article on this...
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Obesity therapeutics update
TweetObesity, 12th century Japan. The Biopharmconsortium Blog has over the years included numerous articles about obesity, and the attempts of researchers and companies to develop treatments for this...
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Vertex cystic fibrosis therapeutics update
Tweet CFTR protein: A. normal B. gating mutant.Source: Lbudd14 http://bit.ly/1rGrzJ1 As we said in our September 10, 2014 article, we intended to post updates on companies that we had been following...
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FDA approves mipomersen (Isis/Genzyme’s Kynamro)–the first...
Tweet Atherosclerosis. Source: Nephron http://bit.ly/jL6Zos In our November 20, 2012 Biopharmconsortium Blog article, entitled “Novel hypercholesterolemia drugs move toward FDA decisions”, we...
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A new wave of small-molecule disease-modifying drugs for cystic fibrosis
Tweet Ivacaftor In our January 24, 2013 article on this blog, we discussed the cases of two genetic diseases, sickle cell disease (SCD) and cystic fibrosis (CF). In both cases, the genetic cause of...
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FDA publishes Draft Guidance on developing drugs for early stages of...
Tweet Normal and Alzheimer’s brains compared. Once again, approaches to improving clinical trials for candidate disease-modifying drugs for Alzheimer’s disease (AD) are in the news. On February 7,...
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Haberman Associates in “Pink Sheet” article on the cystic fibrosis drug market
Tweet Lumacaftor (Vertex’ VX-809) I was quoted in an article in the March 11, 2013 issue of Elsevier Business Intelligence’s The Pink Sheet by senior writer Joseph Haas. The article is entitled...
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Japanese group to take induced pluripotent stem (iPS) cell-derived retinal...
Tweet Stem cells. Source: http://bit.ly/ZnYuFS As reported in Nature News on 27 February 2013 ophthalmologist Masayo Takahashi M.D., Ph.D. and her colleagues at the RIKEN Center for Developmental...
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Gene Therapy Report Published By CHI Insight Pharma Reports
Tweet Adeno-associated virus, a common gene therapy vector. Source: http://bit.ly/1NR7tf4 On November 6, 2015, Cambridge Healthtech Institute (CHI) announced the publication of a new book-length...
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“Our Promise to Nicholas”, Batten disease, and gene therapy
Tweet Wayland MA Source: http://bit.ly/1N1TyRk Russell’s Garden Center, on Route 20, a family-owned business established in 1876, is a unique Wayland MA institution. When you shop at Russell’s and...
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Spark Therapeutics’ retinal disease gene therapy SPK-RPE65 may reach the U.S....
Tweet Spark! Source: http://bit.ly/1Obw4Nk As we discussed in our November 16, 2015 article on this blog, Spark Therapeutics (Philadelphia, PA) recently announced positive top-line results from the...
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Can adoptive cellular immunotherapy successfully treat metastatic...
Tweet Dr. Steven Rosenberg On September 6, 2014, we published an article on this blog announcing the publication of our book-length report, Cancer Immunotherapy: Immune Checkpoint Inhibitors, Cancer...
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Gene therapy for hemophilia—an update
Tweet Tsarevich Alexei of Russia The boy pictured above is Tsarevich Alexei Nikolaevich of Russia, who lived between 1904 and 1918, and was the heir to the throne of Imperial Russia. He is arguably...
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Strimvelis (GSK2696273), a gene therapy for a deadly immunodeficiency in...
TweetAdenosine deaminase Our recent book-length report, Gene Therapy: Moving Toward Commercialization was published by Cambridge Healthtech Institute in November 2015. As indicated by its title, the...
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Obesity therapeutics update
Obesity, 12th century Japan. The Biopharmconsortium Blog has over the years included numerous articles about obesity, and the attempts of researchers and companies to develop treatments for this...
View ArticleClik here to view.
Vertex cystic fibrosis therapeutics update
CFTR protein: A. normal B. gating mutant. Source: Lbudd14 http://bit.ly/1rGrzJ1 As we said in our September 10, 2014 article, we intended to post updates on companies that we had been following on our...
View ArticleClik here to view.
Gene Therapy Report Published By CHI Insight Pharma Reports
Adeno-associated virus, a common gene therapy vector. Source: http://bit.ly/1NR7tf4 On November 6, 2015, Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Gene...
View ArticleClik here to view.
Spark Therapeutics’ retinal disease gene therapy SPK-RPE65 may reach the U.S....
Spark! Source: http://bit.ly/1Obw4Nk As we discussed in our November 16, 2015 article on this blog, Spark Therapeutics (Philadelphia, PA) recently announced positive top-line results from the Phase 3...
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