Roche $4.8bn Buy Sparks Hemophilia Gene Therapy Race
The Swiss major's proposed acquisition of Spark Therapeutics gives it a leading seat at the table in the emerging field of gene therapy.
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Deal Snapshot: Roche gene therapy subsidiary Spark gets option and will partner with start-up NeuExcell on a Huntington’s gene therapy that researchers say may regenerate functional neurons.
Roche’s Phase III of tominersen in Huntington's disease has been stopped after an independent interim review, but with no new or emerging safety signals reported, analysts presume efficacy was the problem.
BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.