Cranbury
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Rocket Pharma stocks up on cardiac gene therapies with $53M Renovacor acquisition
Rocket Pharmaceuticals is acquiring Renovacor, a gene therapies developer whose lead program addresses a particular genetic mutation that leads to a type of heart failure. The all-stock deal gives Rocket the opportunity to expand its reach in genetically driven cardiac diseases.
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PMV’s ASCO data show signs of drugging ‘undruggable’ p53 protein across tumor types
PMV Pharmaceuticals has its first clinical data showing that its lead therapeutic candidate can hit p53, a tumor-suppressing protein long thought to be undruggable. After reporting the preliminary data during ASCO, PMV said it plans to continue the Phase 1 study with the goal of advancing to Phase 2 early next year.
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100% survival in clinical test puts Rocket Pharma gene therapy on course to FDA
A Rocket Pharmaceuticals gene therapy for the rare immune disorder leukocyte adhesion deficiency-I has met the survival goals of a pivotal study, paving the way for regulatory submissions that the company expects to file in early 2023.
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Amicus’s path to unseat Sanofi in rare disease gets steeper after Phase 3 miss
Amicus Therapeutics CEO John Crowley said the totality of data support the Pompe disease therapy’s submission to the FDA. That rolling application is on track for completion in the second quarter.