Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces that patient dosing has commenced in the open-label, Phase 1 clinical trial of RP-A501, the Company’s adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon disease.
Sustained efficacy of ULTOMIRIS was observed on the co-primary endpoints of transfusion avoidance and normalization of lactate dehydrogenase (LDH) levels and the secondary endpoints of LDH level reduction
Under the agreement, Dr. Reddy’s will receive U.S.$70 million as upfront consideration, U.S.$40.5 million in near term milestones and additional financial considerations including existing contractual obligation and inventory.
The program is intended to assist drug supply chain stakeholders, including FDA, in developing the electronic, interoperable system that will identify and trace certain prescription drugs as they
Previously known as COR-001, ziltivekimab is a proprietary anti-interleukin-6 ligand monoclonal antibody (anti-IL6 mAb), targeting residual inflammatory cardiovascular risk in patients living with advanced chronic kidney disease (CKD).
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