CRISPER-Cas9

CRISPR-Cas9 is a new gene-editing technology that has gained enormous press the last few years for it's accuracy.  The science is being used to address the underlying genetic causes of human disease. The advantages seen thus far, is that it is faster, less expensive, and more accurate than prior gene-editing technology.  CRISPR-cas9 enables geneticists and medical researchers to edit parts of the genome, by cutting out, replacing or adding parts to the DNA sequence.  There are a few publicly traded companies.  The companies include the following. 

Intellia Therapeutics:  NTLA
Editas Medicine:  EDIT
Cellectis:  CLLS
Crispr Therapeutics: CRSP

Bottom Line:  The above names are worth following, and learning more about the science of CRISPR-cas9 gene-editing.  Thank you for reading.                

Kalydeco for G551D

Vertex Pharmaceutical's Cystic Fibrosis drug Kalydeco has been FDA approved for people with the G551D mutation since 2012.  Kalydeco has been proven very effective for these patients, as the results from clinical trials reveal.  Below (Vertex 2012 presentation) is sweat chloride, and FEV1 results from the phase 3 G551D clinical trial that led to FDA approval.

 

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The results above at 24 and 48 weeks show extremely sustainable efficacy, versus placebo. Other improvement considerations, would be the dosing of once daily to improve adherence, and bio-availability for oral consumption, with  low or moderate fat containing food.  Kalydeco is currently dosed 150 mg every 12 hours, and prescribed to be taken with fat-containing food. Thank you for reading. 

 

Contact: 586-431-8000                            

VX-661 Corrector for Cystic Fibrosis

Vertex is currently involved with four phase 3 cystic fibrosis clinical trials for people with the F508del mutation, and the combination of potentiator Ivacaftor + corrector VX-661.  Today they gave an update regarding one of the four phase 3 clinical trials that are in progress.  The complete press release is here Vertex Provides Update on Ongoing Phase 3 Program for VX-661 in Combination with Ivacaftor for the Treatment of Cystic Fibrosis.