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Rocket Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on the Pivotal Phase 2 Trial of RP-A501 for the Treatment of Danon Disease
CRANBURY, N.J., August 20, 2025--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease. The hold was lifted in under three months, underscoring the efficiency of the FDA’s review pro Aug 20, 2025 - $RCKT
Gene Therapy Trial Can Restart After Patient Death, FDA Says. The Sector Remains Depressed.
The biotech Rocket Pharmaceuticals said the Food and Drug Administration would allow it to continue a gene therapy trial the agency had put on hold in late May, when a patient in the trial died. In the following months, investor enthusiasm for gene therapies waned even further, as what was once the biggest idea in biotech delivered even more disappointments and safety worries. Rocket’s relatively quick reprieve is good news for the stock, and suggests that the FDA remains actively supportive of gene therapy development. Aug 20, 2025 - $RCKT
Jefferies Raises PT on Rocket Pharmaceuticals, Inc. (RCKT) to $3.00; Maintains ‘Hold’ Rating
With significant hedge fund interest and a share price under $10, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) secures a spot on our list of the 11 Best Single Digit Stocks to Buy According to Hedge Funds. On August 8, 2025, Jefferies increased its price target on Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) from $2.50 to $3.00, maintaining a ‘Hold’ […] Aug 19, 2025 - $RCKT
Rocket Pharmaceuticals Reorganizes, Cuts Workforce by 30% to Focus on AAV Cardiovascular Platform
Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is one of the most promising penny stocks under $5. On July 24, Rocket Pharmaceuticals announced a strategic corporate reorganization and pipeline prioritization to focus its resources on its adeno-associated virus/AAV cardiovascular platform and the regulatory submission for KRESLADI (formerly RP-L201) for severe leukocyte adhesion deficiency-I/LAD-I. The company is implementing a […] Aug 18, 2025 - $RCKT
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