Rocket Pharmaceuticals(RCKT)

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RCKT Stock Up as FDA Accepts Resubmitted BLA for Gene Therapy
ZACKS· 2025-10-15 16:35
Key Takeaways RCKT's resubmitted BLA for Kresladi to treat severe LAD-I has been accepted by the FDA.FDA's decision is expected by March 28, 2026, with potential approval marking RCKT's first product.The resubmission follows an earlier FDA CRL for Kresladi BLA issued to RCKT last June.Rocket Pharmaceuticals (RCKT) announced that the FDA has accepted the resubmitted biologics license application (BLA) seeking approval for its gene therapy Kresladi (marnetegragene autotemcel; marne-cel) to treat patients with ...
FDA Acceptance Of Rocket Pharmaceuticals' Lead Gene Therapy Application Reestablishes Momentum For Stock
Benzinga· 2025-10-14 18:27
On Tuesday, the U.S. Food and Drug Administration (FDA) accepted Rocket Pharmaceuticals, Inc.’s (NASDAQ:RCKT) resubmission of the Biologics License Application (BLA) for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).LAD-I is a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem ...
Domino's Pizza Posts Upbeat Results, Joins Astria Therapeutics, Albertsons Companies, Polaris And Other Big Stocks Moving Higher On Tuesday - American Resources (NASDAQ:AREC), Albertsons Companies (NY
Benzinga· 2025-10-14 14:15
Group 1: U.S. Stock Market Overview - U.S. stocks experienced a decline, with the Dow Jones index dropping approximately 300 points on Tuesday [1] Group 2: Domino's Pizza, Inc. Performance - Domino's Pizza reported third-quarter earnings per share of $4.08, surpassing the analyst consensus estimate of $3.96 [1] - The company's quarterly sales reached $1.147 billion, reflecting a year-over-year increase of 6.2%, exceeding the expected $1.137 billion [1] - Following the positive financial results, Domino's shares increased by 4.1% to $424.95 on Tuesday [1] Group 3: Notable Stock Gains - Astria Therapeutics, Inc. shares surged 40.6% to $11.91 after BioCryst announced plans to acquire the company [4] - Critical Metals Corp. shares rose 22.9% to $28.62 amid U.S.-China trade tensions and JPMorgan's $1.5 trillion initiative [4] - Trilogy Metals Inc. gained 20.6% to $7.93 [4] - American Resources Corporation jumped 17.5% to $5.85, participating in additional October conferences [4] - Navitas Semiconductor Corporation rose 16.3% to $11.60 after providing a progress update on its power devices [4] - Telefonaktiebolaget LM Ericsson gained 16% to $9.47, reporting third-quarter EPS and sales above estimates [4] - Rocket Pharmaceuticals, Inc. increased by 15.5% to $4.0100 after FDA acceptance of its Biologics License Application resubmission [4] - Orla Mining Ltd. surged 11.2% to $12.74, reporting strong gold production from Musselwhite [4] - Albertsons Companies, Inc. rose 9.7% to $18.58 after better-than-expected second-quarter results and a $750 million share repurchase agreement [4] - Polaris Inc. gained 9.2% to $66.98, announcing the divestment of its majority stake in Indian Motorcycle [4] - Spyre Therapeutics, Inc. rose 11.2% to $21.04, pricing a $275 million public offering of common stock [4]
Rocket Pharma says gene therapy gets FDA review (RCKT:NASDAQ)
Seeking Alpha· 2025-10-14 12:21
Rocket Pharmaceuticals (NASDAQ:RCKT) added ~11% in the premarket on Tuesday after the company announced that the FDA had accepted its resubmitted marketing application for its gene therapy candidate Kresladi. In June 2024, the FDA rejected the company’s biologics license application for ...
Rocket Pharmaceuticals withdraws review application to FDA for RP-L102
Seeking Alpha· 2025-10-03 16:22
Rocket Pharmaceuticals (NASDAQ:RCKT) reported that it has voluntarily withdrawn its biologics marketing application to the U.S. FDA for mozafancogene autotemcel or RP-L102, an investigational gene therapy for the treatment of Fanconi Anemia. This decision follows the company’s previously announced corporate ...
Rocket Pharmaceuticals withdraws US application of gene therapy for rare blood disorder
Reuters· 2025-10-03 11:11
Rocket Pharmaceuticals said on Friday that it has withdrawn its application for U.S approval of its experimental gene therapy for a rare inherited blood disorder. ...
Kuehn Law Encourages Investors of Rocket Pharmaceuticals, Inc. to Contact Law Firm
Prnewswire· 2025-09-19 17:30
Core Viewpoint - Kuehn Law, PLLC is investigating potential breaches of fiduciary duties by certain officers and directors of Rocket Pharmaceuticals, Inc. [1] Company Summary - Rocket Pharmaceuticals, Inc. is currently under scrutiny for possible misconduct by its leadership, which may affect shareholder interests [1]
Rocket Pharmaceuticals, Inc. (RCKT) Presents at Morgan Stanley 23rd
Seeking Alpha· 2025-09-09 16:36
PresentationMichael UlzEquity Analyst All right. Good morning, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference. I'm Mike Ulz, one of the biotech analysts here. And it's my pleasure to introduce Gaurav Shah, CEO of Rocket Pharmaceuticals. Before we get started, I just need to read a quick disclosure. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. And if you have any questions, please rea ...
Rocket Pharmaceuticals, Inc. (RCKT) Presents At Morgan Stanley 23rd Annual Global Healthcare Conference Transcript
Seeking Alpha· 2025-09-09 16:36
PresentationMichael UlzEquity Analyst All right. Good morning, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference. I'm Mike Ulz, one of the biotech analysts here. And it's my pleasure to introduce Gaurav Shah, CEO of Rocket Pharmaceuticals. Before we get started, I just need to read a quick disclosure. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. And if you have any questions, please rea ...
Rocket Pharmaceuticals (NasdaqGM:RCKT) FY Conference Transcript
2025-09-09 14:15
Summary of Rocket Pharmaceuticals FY Conference Call Company Overview - **Company**: Rocket Pharmaceuticals (NasdaqGM:RCKT) - **Industry**: Gene Therapy - **Focus**: Development of gene therapies for rare diseases, particularly in cardiomyopathy and hematology Key Points Company and Product Pipeline - Rocket Pharmaceuticals operates as a dual platform company with both in vivo AAV-based cardiac programs and ex vivo lentiviral-based hematology programs in development [2][3] - The company has six disclosed programs, including three cardiac programs targeting Danon Disease, PKP2-arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy, collectively affecting at least 100,000 patients in the U.S. and Europe [2][3] - The lentiviral portfolio includes Leukocyte Adhesion Deficiency-I, Fanconi Anemia, and Pyruvate Kinase Deficiency, with a decision to pause new spending on the latter two to focus resources on AAV-based cardiac programs [3][4] Financial and Operational Updates - A workforce reduction was implemented to prioritize certain programs and extend cash runway, with cash expected to last into Q2 2027 [4] - The company aims to be first, best, and only in class for its gene therapy products, targeting diseases with increasing market sizes [3][4] Clinical Trials and Regulatory Interactions - The pivotal phase two trial for Danon Disease was put on clinical hold due to increased risk of TMA (Thrombotic Microangiopathy) but was lifted in less than three months after modifications to the treatment regimen [9][10] - The trial will start in early 2026, with a three-month troponin run-in period before patient treatment begins [10] - The pivotal trial design includes a 12-patient trial with composite endpoints of protein expression and LV mass index reductions, agreed upon with the FDA [24][30] Market Opportunity - The prevalence of Danon Disease is estimated at 15,000 to 30,000 patients in the U.S. and Europe, with ongoing efforts to identify more patients through genetic testing [34] - The company is positioned to potentially file for commercialization in the next few years, contingent on trial outcomes [35][36] Future Catalysts - Upcoming catalysts include the PDUFA date for CRISLATI, clarity on PKP2 trial design, and updates on the Danon program and BAG3 phase one start [61][62] - The company plans to proceed cautiously with its programs, focusing on one at a time to ensure successful execution [62] Additional Insights - The company has learned valuable lessons from the Danon Disease program that will inform future trials, particularly regarding safety monitoring and regulatory interactions [38][39] - The choice of vectors for gene therapy is strategic, with AAV9 used for Danon Disease and RH74 for PKP2 and BAG3, based on regulatory experience and patient community feedback [55] Conclusion - Rocket Pharmaceuticals is actively advancing its gene therapy programs with a focus on rare diseases, navigating regulatory challenges, and preparing for future commercialization opportunities while ensuring patient safety and efficacy in its clinical trials [61][62]