Laura Andrews

TL;DR: A comparison of the two recombinant protein therapeutics, Fabrazyme and Replagal, found that both drugs appear to be functionally indistinguishable, and the data from these studies provide no rationale for the use of these proteins at different therapeutic doses.

TL;DR: These studies found AAV2-sFLT01 to be well-tolerated, localized, and capable of long-term expression.

TL;DR: In a mouse model of Pompe disease, glycogen was more efficiently removed from the heart than from skeletal muscle for all enzymes, and overall, the CHO cell-derived rhGAA reduced glycogen to a greater extent than that observed with the other enzymes.

TL;DR: The addendum of ICHS6 provides a timely opportunity for the scientific and regulatory community to embrace strategies which minimize primate use and increase efficiency of mAb development.

TL;DR: Alternative approaches, including animal models of disease, genetically modified mice, or use of surrogate molecules, may improve the predictive value of preclinical safety assessments of species-specific biopharmaceuticals, although many caveats associated with these models must be considered.