Biotech Strategy Blog

Commentary on Science, Innovation & New Products

Posts tagged ‘Vertex’

Letter from Boston

I recently returned from a few days in Boston & Cambridge, so today, in memory of the late Alastair Cooke and his Letter from America, broadcast for 58 years from 1946 to 2004, I wanted to share with you my “Letter from Boston”.

New England is the No 1 biotechnology region on the East Coast of the United States and the Boston/Cambridge area of Massachusetts is the hub.

What makes Boston/Cambridge so attractive as a biotech region?  Amongst many, I’d suggest 3 factors stand out to me:

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Vertex plans NDA for VX-770 in Cystic Fibrosis for Second Half of 2011

As an update to this morning’s blog post that mentioned Vertex’s VX-770, the company have just announced their key business objectives for 2011.  Further information will be included in the presentation by Vertex at the JP Morgan Healthcare conference scheduled for later today.

The news in Cystic Fibrosis is that if the phase 3 clinical trial data is positive the NDA for VX-770 is expected in the second half of 2011.  The following are the relevant sections from the press release:

Cystic Fibrosis: Phase 3 Registration Program for VX-770 Nears Completion

VX-770 NDA Submission Planned for Second Half of 2011

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New research points to role of Cystic Fibrosis Transmembrane Conductance Regulator Signaling in Emphysema

One of the exciting things about the biotechnology industry is its ability to innovate and translate developments in basic science into potential new drugs.

I previously wrote about denufosol in cystic fibrosis (CF), a disease that affects about 30,000 people in the United States and 70,000 worldwide.  The disease is characterized by the accumulation of mucus that leads to bacterial overgrowth and chronic lung infections. Mucus cannot be removed from the lung in CF due to abnormal mucociliary transport resulting from impaired epithelial chloride secretion and sodium hyperabsorption.  This is now known to be due to defective cystic fibrosis transmembrane regulator (CFTR) protein. A good overview of this can be found in the 2006 New England Journal of Medicine Editorial by Felix Ratjen, “Restoring Airway Surface Liquid in Cystic Fibrosis.”

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