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Rocket Pharmaceuticals Inc

Rocket Pharmaceuticals, Inc. is a late-stage biotechnology company that develops, manufactures, and sells genetic therapies for rare and devastating diseases in the United States. Its in vivo adeno-associated viral (AAV) programs include RP-A501 for Danon disease in Phase 2, RP-A601 for Plakophilin-2 Arrhythmogenic Cardiomyopathy in Phase 1, and a preclinical program for BAG3 Dilated Cardiomyopathy. Its ex vivo lentiviral (LV) programs include RP-L201 for Leukocyte Adhesion Deficiency-I, RP-L102 for Fanconi Anemia, and RP-L301 for Pyruvate Kinase Deficiency. The company has license agreements with several research institutions and universities, and was founded in 1999 and is headquartered in Cranbury, New Jersey.

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Biotech & Genomic Medicine

Rocket Pharmaceuticals Completes $180 Million Priority Review Voucher Sale

Rocket Pharmaceuticals completed the sale of its Rare Pediatric Disease Priority Review Voucher for gross proceeds of $180 million. The voucher was awarded by the FDA in March 2026 following the approval of Kresladi, a gene therapy for severe leukocyte adhesion deficiency-I. Before the transaction, the company reported cash, cash equivalents, and investments of $144.4 million as of March 31, 2026, and the non-dilutive proceeds increased its pro forma cash position to approximately $322.6 million, extending its expected operational runway through the second quarter of 2028. BofA Securities analyst Jason Zemansky recently raised the price target on Rocket Pharmaceuticals to $10 from $9 and maintained a Buy rating, citing the restart of RP-A501 dosing as a positive step in the company's near-term recovery strategy.
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