Re: Dr. Tran of Providence hired by AZN | Already at AZN 3+ months ago
''In time and with money, as with any problem at hand, with so many resources, eventually CRSPR might emerge as a viable tool for curing human diseases.''
CRSP's exagamglogene autotemcel is essentially a functional cure for sickle cell disease and transplant dependent beta thalassemia. It should be approved by the FDA sometime next year. Thankfully, they and many other companies have a range of ex vivo and in vivo therapies in the clinic.
''For the next three years, I will rest on Dr. Deniger's assessment of SB -- simple, economic vectoring.''
Well, he is hardly going to say it can't precisely edit or be used as a screening tool for various enhancing edits and identify mechanisms of response/resistance.