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.
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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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.
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.
The company acquired 10 AAV-based gene therapies in development for lysosomal storage disorders, including two already in early clinical trials.