Introduction
Today’s advanced therapies target devastating diseases with high unmet need. Given the urgent medical need (and pressure from patient advocates), regulators increasingly must make decisio…
Successes in gene therapy for hemophilia B and A
Hemophilia A and B are X-linked genetic diseases which prevents the formation of functional coagulant factors VII and IX respectively and cause a prope…
A hammer finds new nails (which happen to be eyeballs)
The insulin growth factor receptor 1 (IGF-1R) was once upon a time a popular cancer target pursued by multiple biopharmas each with their own hum…
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