Re: Monogenetic disorders and their potential...
From Slide 11 in the Leerink Presentation...
Hemoglobinopathies(SCA, B-thalassemia)
Immune System Disorders (X-SCID, ADA-SCID, W-A)
Liposomal Storage Diseases(Gaucher, Fabry, Pompe)
Sangamo's 8K disclosed that Liposomal Storage Diseases were part of the Shire Agreement. Several press releases indicate simply that Shire has rights to Factor 7, 8, 9 and 10, as well as three additional gene targets. Shire seems to want to keep confidential the additional gene targets for competitive reasons, however, clues have been slowly leaking thru presentations and announcements and investment seminars featuring EL and Sangamo. I believe the three additional gene targets are included in slide 11 and are: Liposomal Storage Diseases(Gaucher, Fabry, Pompe). Shire is already active in the Liposomal treatment market and most probably have included these disorders in their additional three gene targets.
What will be the first functional cure announcement out of the Shire Partnership? At first I thought it would be Hemophilia B, however due to the confidentiality around the three additional gene targets I now believe that one of these three diseases will be the first functional cure from this partnership. Anyone care to guess which it will be? Gaucher, Fabry, Pompe???
When the announcement is made that Gaucher disease has been cured in laboratory mice and confirms the Hemophilia B proof of concept... what will the share price do on that date in 2012 or 2013.
Will we see a 25% surge in share price from an $8 per share level? Perhaps a move from $8 to $10 in one day...
Or will we see a move from $10 to $20 due to the "proof of concept" and validation of the $1.5 Billion + shire deal?
Great times are ahead of us if and when this day comes...
JMHO,
RM
